Publications

Background

Robust expansion of human hepatocytes in Fah-/- / Rag2-/- / Il2rg-/- mice. – Azuma H, Paulk N, Ranade A, Dorrell C, Al-Dhalimy M, Ellis E, Strom S, Kay MA, Finegold M, Grompe M. Nat Biotechnol. 2007 Aug;25(8):903-10
Extensive double humanization of both liver and hematopoiesis in FRGN mice. – Wilson EM, Bial J, Tarlow B, Bial G, Jensen B, Greiner DL, Brehm MA, Grompe M. Stem Cell Res. 2014 Nov;13(3 Pt A):404-12.
Fah Knockout Animals as Models for Therapeutic Liver Repopulation. – M Grompe. Adv Exp Med Biol. 2017;959:215-230.
Successful Engraftment of Human Hepatocytes in uPA-SCID and FRG® KO Mice. – Foquet L, Wilson EM, Verhoye L, Grompe M, Leroux-Roels G, Bial J, Meuleman P. Methods Mol Biol. 2017;1506:117-130
Mice with human livers. – Grompe M, Strom SC. Gastroenterology. 2013 Dec;145(6):1209-14
Intrablastocyst injection with human CD34+/CD133+ cells increase survival of immunocompetent fumarylacetoacetate hydrolase knockout mice. – Marra E, Turrini P, Tripodi M, Ciliberto G, Padron J, Aurisicchio L. Lab Anim. 2012 Oct;46(4):280-6.
Xeno-repopulation of Fah-/- Nod/Scid mice livers by human hepatocytes. – Su B, Liu C, Xiang D, Zhang H, Yuan S, Wang M, Chen F, Zhu H, He Z, Wang X, Hu Y. Sci China Life Sci. 2011 Jun;54(3):227-34
Liver xeno-repopulation with human hepatocytes in Fah-/-Rag2-/- mice after pharmacological immunosuppression. He Z, Zhang H, Zhang X, Xie D, Chen Y, Wangensteen KJ, Ekker SC, Firpo M, Liu C, Xiang D, Zi X, Hui L, Yang G, Ding X, Hu Y, Wang X. Am J Pathol. 2010 Sep;177(3):1311-9.
Nucleotide sequence of a cDNA encoding murine fumarylacetoacetate hydrolase. – Grompe M, al-Dhalimy M. Biochem Med Metab Biol. 1992 Aug;48(1):26-31.

Gene Therapy & Gene Editing

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model. – Lisowski L, Dane AP, Chu K, Zhang Y, Cunningham SC, Wilson EM, Nygaard S, Grompe M, Alexander IE, Kay MA. Nature. 2014 Feb 20;506(7488):382-6.
Methods Matter: Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct Vectors. Rumachik NG, Malaker SA, Poweleit N, Maynard LH, Adams CM, Leib RD, Cirolia G, Thomas D, Stamnes S, Holt K, Sinn P, May AP, Paulk NK. Mol Ther Methods Clin Dev. 2020 May 22;18:98-118.
Restoring the natural tropism of AAV2 vectors for human liver. Cabanes-Creus M, Hallwirth CV, Westhaus A, Ng BH, Liao SHY, Zhu E, Navarro RG, Baltazar G, Drouyer M, Scott S, Logan GJ, Santilli G, Bennett A, Ginn SL, McCaughan G, Thrasher AJ, Agbandje-McKenna M, Alexander IE, Lisowski L. Sci Transl Med. 2020 Sep 9;12(560):eaba3312.
Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity. – Paulk NK, Pekrun K, Zhu E, Nygaard S, Li B, Xu J, Chu K, Leborgne C, Dane AP, Haft A, Zhang Y, Zhang F, Morton C, Valentine MB, Davidoff AM, Nathwani AC, Mingozzi F, Grompe M, Alexander IE, Lisowski L, Kay MA. Mol Ther. 2018 Jan 3;26(1):289-303.
Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice. Zou C, Vercauteren KOA, Michailidis E, Kabbani M, Zoluthkin I, Quirk C, Chiriboga L, Yazicioglu M, Anguela XM, Meuleman P, High KA, Herzog RW, de Jong YP. Mol Ther Methods Clin Dev. 2020 Jun 2;18:189-198.
Superior human hepatocyte transduction with adeno-associated virus vector serotype 7. – Shao W, Pei X, Cui C, Askew C, Dobbins A, Chen X, Abajas YL, Gerber DA, Samulski RJ, Nichols TC, Li C. Gene Ther. 2019 Sep 30.
Using a barcoded AAV capsid library to select for clinically relevant gene therapy vectors. Pekrun K, De Alencastro G, Luo QJ, Liu J, Kim Y, Nygaard S, Galivo F, Zhang F, Song R, Tiffany MR, Xu J, Hebrok M, Grompe M, Kay MA. JCI Insight. 2019 Nov 14;4(22). pii: 131610.
Somatic Liver Knockout (SLiK): A Quick and Efficient Way to Generate Liver-Specific Knockout Mice Using Multiplex CRISPR/Cas9 Gene Editing. Johnson CG, Chen T, Furey N, Hemmingsen MG, Bissig KD. Curr Protoc Mol Biol. 2020 Mar;130(1):e117.
High throughput in vitro, ex vivo and in vivo screen of AAV vectors based on physical and functional transduction. Westhaus A, Cabanes Creus M, Rybicki A, Baltazar G, Navarro RG, Zhu E, Drouyer M, Knight M, Albu RF, Ng BH, Kalajdzic P, Kwiatek M, Hsu K, Santilli G, Gold W, Kramer B, Gonzalez-Cordero A, Thrasher AJ, Alexander IE, Lisowski L. Hum Gene Ther. 2020 Jan 30.
AAV-Mediated Gene Delivery to the Mouse Liver. Cunningham SC, Alexander IE. Methods Mol Biol. 2019;1937:213-219.
Tracking adeno-associated virus capsid evolution by high-throughput sequencing. de Alencastro G, Pekrun K, Valdmanis P, Tiffany M, Xu J, Kay MA. Hum Gene Ther. 2020 Feb 5.
Partial pancreatic transdifferentiation of primary human hepatocytes in the livers of a humanised mouse model. – Ren B, La QT, O’Brien BA, Nassif NT, Tan Y, Gerace D, Martiniello-Wilks R, Torpy F, Dane AP, Alexander IE, Simpson AM. J Gene Med. 2018 May;20(5):e3017.
Curative Ex Vivo Hepatocyte-Directed Gene Editing in a Mouse Model of Hereditary Tyrosinemia Type 1. – VanLith C, Guthman R, Nicolas CT, Allen K, Du Z, Joo DJ, Nyberg SL, Lillegard JB, Hickey RD. Hum Gene Ther. 2018 Nov;29(11):1315-1326.
CRISPR/Cas9: at the cutting edge of hepatology. – Pankowicz FP, Jarrett KE, Lagor WR, Bissig KD. Gut. 2017 Jul;66(7):1329-1340.
Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality. Cabanes-Creus M, Ginn SL, Amaya AK, Liao SHY, Westhaus A, Hallwirth CV, Wilmott P, Ward J, Dilworth KL, Santilli G, Rybicki A, Nakai H, Thrasher AJ, Filip AC, Alexander IE, Lisowski L. Mol Ther Methods Clin Dev. 2018 Nov 1;12:71-84.
Identification of liver-specific enhancer-promoter activity in the 3′ untranslated region of the wild-type AAV2 genome. Logan GJ, Dane AP, Hallwirth CV, Smyth CM, Wilkie EE, Amaya AK, Zhu E, Khandekar N, Ginn SL, Liao SHY, Cunningham SC, Sasaki N, Cabanes-Creus M, Tam PPL, Russell DW, Lisowski L, Alexander IE. Nat Genet. 2017 Aug;49(8):1267-1273.
Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects. Baruteau J, Waddington SN, Alexander IE, Gissen P. J Inherit Metab Dis. 2017 Jul;40(4):497-517.
CRISPR/Cas9 – Mediated Precise Targeted Integration In Vivo Using a Double Cut Donor with Short Homology Arms. – Yao X, Wang X, Liu J, Hu X, Shi L, Shen X, Ying W, Sun X, Wang X, Huang P, Yang H. EBioMedicine. 2017 Jun;20:19-26.
Delivering efficient liver-directed AAV-mediated gene therapy. Baruteau J, Waddington SN, Alexander IE, Gissen P. Gene Ther. 2017 May;24(5):263-264.
AAV-Mediated Gene Delivery to the Liver: Overview of Current Technologies and Methods. Palaschak B, Herzog RW, Markusic DM. Methods Mol Biol. 2019;1950:333-360.
A universal system to select gene-modified hepatocytes in vivo. – Nygaard S, Barzel A, Haft A, Major A, Finegold M, Kay MA, Grompe M. Sci Transl Med. 2016 Jun 8;8(342):342ra79.
Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid. – Vercauteren K, Hoffman BE, Zolotukhin I, Keeler GD, Xiao JW, Basner-Tschakarjan E, High KA, Ertl HC, Rice CM, Srivastava A, de Jong YP, Herzog RW. Mol Ther. 2016 Jun;24(6):1042-1049.
Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids. – Wang L, Bell P, Somanathan S, Wang Q, He Z, Yu H, McMenamin D, Goode T, Calcedo R, Wilson JM. Mol Ther. 2015 Dec;23(12):1877-87.
Adeno-associated virus serotypes for gene therapeutics. Lisowski L, Tay SS, Alexander IE. Curr Opin Pharmacol. 2015 Oct;24:59-67.
Efficient genetic manipulation of the NOD-Rag1-/-IL2RgammaC-null mouse by combining in vitro fertilization and CRISPR/Cas9 technology. – Li F, Cowley DO, Banner D, Holle E, Zhang L, Su L. Sci Rep. 2014 Jun 17;4:5290.
Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype. – Yin H, Xue W, Chen S, Bogorad RL, Benedetti E, Grompe M, Koteliansky V, Sharp PA, Jacks T, Anderson DG. Nat Biotechnol. 2014 Jun;32(6):551-3.
AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence. – Wang Z, Lisowski L, Finegold MJ, Nakai H, Kay MA, Grompe M. Mol Ther. 2012 Oct;20(10):1902-11.
AAV-mediated gene targeting is significantly enhanced by transient inhibition of nonhomologous end joining or the proteasome in vivo. – Paulk NK, Loza LM, Finegold MJ, Grompe M. Hum Gene Ther. 2012 Jun;23(6):658-65.
Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo. – Paulk NK, Wursthorn K, Wang Z, Finegold MJ, Kay MA, Grompe M. Hepatology. 2010 Apr;51(4):1200-8
Chromosomal integration of adenoviral vector DNA in vivo. – Stephen SL, Montini E, Sivanandam VG, Al-Dhalimy M, Kestler HA, Finegold M, Grompe M, Kochanek S. J Virol. 2010 Oct;84(19):9987-94.

Infectious Diseases: Malaria

Human Antibodies that Slow Erythrocyte Invasion Potentiate Malaria-Neutralizing Antibodies. – Alanine DGW, Quinkert D, Kumarasingha R, Mehmood S, Donnellan FR, Minkah NK, Dadonaite B, Diouf A, Galaway F, Silk SE, Jamwal A, Marshall JM, Miura K, Foquet L, Elias SC, Labbé GM, Douglas AD, Jin J, Payne RO, Illingworth JJ, Pattinson DJ, Pulido D, Williams BG, de Jongh WA, Wright GJ, Kappe SHI, Robinson CV, Long CA, Crabb BS, Gilson PR, Higgins MK, Draper SJ. Cell. 2019 Jun 27;178(1):216-228.e21.
A comprehensive model for assessment of liver stage therapies targeting Plasmodium vivax and Plasmodium falciparum. – Roth A, Maher SP, Conway AJ, Ubalee R, Chaumeau V, Andolina C, Kaba SA, Vantaux A, Bakowski MA, Thomson-Luque R, Adapa SR, Singh N, Barnes SJ, Cooper CA, Rouillier M, McNamara CW, Mikolajczak SA, Sather N, Witkowski B, Campo B, Kappe SHI, Lanar DE, Nosten F, Davidson S, Jiang RHY, Kyle DE, Adams JH. Nat Commun. 2018 May 9;9(1):1837.
A Humanized Mouse Model for Plasmodium vivax to Test Interventions that Block Liver Stage to Blood Stage Transition and Blood Stage Infection. Schäfer C, Roobsoong W, Kangwanrangsan N, Bardelli M, Rawlinson TA, Dambrauskas N, Trakhimets O, Parthiban C, Goswami D, Reynolds LM, Kennedy SY, Flannery EL, Murphy SC, Sather DN, Draper SJ, Sattabongkot J, Mikolajczak SA, Kappe SHI. iScience. 2020 Aug 21;23(8):101381.
A human monoclonal antibody prevents malaria infection and defines a new site of vulnerability on Plasmodium falciparum circumsporozoite protein. – Kisalu NK, Idris AH, Weidle C, Flores-Garcia Y, Flynn BJ, Sack BK, Murphy S, Schön A, Freire E, Francica JR, Miller AB, Gregory J, March S, Liao HX, Haynes BF, Wiehe K, Trama AM, Saunders KO, Gladden MA, Monroe A, Bonsignori M, Kanekiyo M, Wheatley AK, McDermott AB, Farney SK, Chuang GY, Zhang B, Kc N, Chakravarty S, Kwong PD, Sinnis P, Bhatia SN, Kappe SHI, Sim BKL, Hoffman SL, Zavala F, Pancera M, Seder RA. Nat Med. 2018 May;24(4):408-416.
A Plasmodium berghei sporozoite-based vaccination platform against human malaria. – Mendes AM, Machado M, Gonçalves-Rosa N, Reuling IJ, Foquet L, Marques C, Salman AM, Yang ASP, Moser KA, Dwivedi A, Hermsen CC, Jiménez-Díaz B, Viera S, Santos JM, Albuquerque I, Bhatia SN, Bial J, Angulo-Barturen I, Silva JC, Leroux-Roels G, Janse CJ, Khan SM, Mota MM, Sauerwein RW, Prudêncio M. NPJ Vaccines. 2018 Aug 24;3:33.
Genetic mapping of fitness determinants across the malaria parasite Plasmodium falciparum life cycle. Li X, Kumar S, McDew-White M, Haile M, Cheeseman IH, Emrich S, Button-Simons K, Nosten F, Kappe SHI, Ferdig MT, Anderson TJC, Vaughan AM. PLoS Genet. 2019 Oct 14;15(10):e1008453.
A recombinant antibody against Plasmodium vivax UIS4 for distinguishing replicating from dormant liver stages. – Schafer C, Dambrauskas N, Steel RW, Carbonetti S, Chuenchob V, Flannery EL, Vigdorovich V, Oliver BG, Roobsoong W, Maher SP, Kyle D, Sattabongkot J, Kappe SHI, Mikolajczak SA, Sather DN. Malar J. 2018 Oct 17;17(1):370.
Assessing drug efficacy against Plasmodium falciparum liver stages in vivo. – Flannery EL, Foquet L, Chuenchob V, Fishbaugher M, Billman Z, Navarro MJ, Betz W, Olsen TM, Lee J, Camargo N, Nguyen T, Schafer C, Sack BK, Wilson EM, Saunders J, Bial J, Campo B, Charman SA, Murphy SC, Phillips MA, Kappe SH, Mikolajczak SA. JCI Insight. 2018 Jan 11;3(1).
Characterization of Plasmodium vivax Proteins in Plasma-Derived Exosomes From Malaria-Infected Liver-Chimeric Humanized Mice. – Gualdrón-López M, Flannery EL, Kangwanrangsan N, Chuenchob V, Fernandez-Orth D, Segui-Barber J, Royo F, Falcón-Pérez JM, Fernandez-Becerra C, Lacerda MVG, Kappe SHI, Sattabongkot J, Gonzalez JR, Mikolajczak SA, Del Portillo HA. Front Microbiol. 2018 Jun 25;9:1271.
Humanized Mice Are Instrumental to the Study of Plasmodium falciparum Infection. – Tyagi RK, Tandel N, Deshpande R, Engelman RW, Patel SD, Tyagi P. Front Immunol. 2018 Dec 13;9:2550.
Humanized Mouse Models for the Study of Human Malaria Parasite Biology, Pathogenesis, and Immunity. – Minkah NK, Schafer C, Kappe SHI. Front Immunol. 2018 Apr 19;9:807.
Plasmodium falciparum Liver Stage Infection and Transition to Stable Blood Stage Infection in Liver-Humanized and Blood-Humanized FRGN KO Mice Enables Testing of Blood Stage Inhibitory Antibodies (Reticulocyte-Binding Protein Homolog 5) In Vivo. – Foquet L, Schafer C, Minkah NK, Alanine DGW, Flannery EL, Steel RWJ, Sack BK, Camargo N, Fishbaugher M, Betz W, Nguyen T, Billman ZP, Wilson EM, Bial J, Murphy SC, Draper SJ, Mikolajczak SA, Kappe SHI. Front Immunol. 2018 Mar 14;9:524.
Humoral protection against mosquito bite-transmitted Plasmodium falciparum infection in humanized mice. – Sack BK, Mikolajczak SA, Fishbaugher M, Vaughan AM, Flannery EL, Nguyen T, Betz W, Jane Navarro M, Foquet L, Steel RWJ, Billman ZP, Murphy SC, Hoffman SL, Chakravarty S, Sim BKL, Behet M, Reuling IJ, Walk J, Scholzen A, Sauerwein RW, Ishizuka AS, Flynn B, Seder RA, Kappe SHI. NPJ Vaccines. 2017 Oct 9;2:27.
Diversity-oriented synthesis yields novel multistage antimalarial inhibitors. – Kato N, Comer E, Sakata-Kato T, Sharma A, Sharma M, Maetani M, Bastien J, Brancucci NM, Bittker JA, Corey V, Carke D, Derbyshire ER, Dornan GL, Duffy S, Eckley S, Itoe MA, Koolen KM, Lewis TA, Lui PS, Lukens AK, Lund E, March S, Meibalan E, Meier BC, McPhail JA, Mitasev B, Moss EL, Sayes M, Van Gessel Y, Wawer MJ, Yoshinaga T, Zeeman AM, Avery VM, Bhatia SN, Burke JE, Catteruccia F, Clardy JC, Clemons PA, Dechering KJ, Duvall JR, Foley MA, Gusocsky F, Kocken CH, Marti M, Morningstar ML, Munoz B, Neafsey DE, Sharma A, Winzeler EA, Wirth DF, Scherer CA, Schreiber SL. Nature. 2016 Oct 20;538(7625):344-349
Host-based Prophylaxis Successfully Targets Liver Stage Malaria Parasites. – Douglass AN, Kain HS, Abdullahi M, Arang N, Austin LS, Mikolajczak SA, Billman ZP, Hume JCC, Murphy SC, Kappe SHI, Kaushansky A. Mol Ther. 2015 May;23(5):857-865.
Malaria vaccines: identifying Plasmodium falciparum liver-stage targets. – Longley RJ, Hill AV, Spencer AJ. Front Microbiol. 2015 Sep 15;6:965.
Plasmodium vivax liver stage development and hypnozoite persistence in human liver-chimeric mice. – Mikolajczak SA, Vaughan AM, Kangwanrangsan N, Roobsoong W, Fishbaugher M, Yimamnuaychok N, Rezakhani N, Lakshmanan V, Singh N, Kaushansky A, Camargo N, Baldwin M, Lindner SE, Adams JH, Sattabongkot J, Kappe SH. Cell Host Microb. 2015 Apr 8;17(4):526-35
Of men in mice: the success and promise of humanized mouse models for human malaria parasite infections. – Kaushansky A, Mikolajczak SA, Vignali M, Kappe SH. Cell Microbiol. 2014 May;16(5):602-11.
Molecular detection and quantification of Plasmodium falciparum-infected human hepatocytes in chimeric immune-deficient mice. – Foquet L, Hermsen CC, van Gemert GJ, Libbrecht L, Sauerwein R, Meuleman P, Leroux-Roels G. Malar J. 2013 Nov 24;12:430.
A transgenic Plasmodium falciparum NF54 strain that expresses GFP-luciferase throughout the parasite life cycle. – Vaughan AM, Mikolajczak SA, Camargo N, Lakshmanan V, Kennedy M, Lindner SE, Miller JL, Hume JC, Kappe SH. Mol Biochem Parasitol. 2012 Dec;186(2):143-7.
Comprehensive proteomics investigation of P. vivax-infected human plasma and parasite isolates. Venkatesh A, Aggarwal S, Kumar S, Rajyaguru S, Kumar V, Bankar S, Shastri J, Patankar S, Srivastava S. BMC Infect Dis. 2020 Mar 2;20(1):188.
Complete Plasmodium falciparum liver-stage development in liver-chimeric mice. – Vaughan AM, Mikolajczak SA, Wilson EM, Grompe M, Kaushansky A, Camargo N, Bial J, Ploss A, Kappe SH. J Clin Invest. 2012 Oct 1;122(10):3618-28
Development of humanized mouse models to study human malaria parasite infection. – Vaughan AM, Kappe SH, Ploss A, Mikolajczak SA. Future Microbiol. 2012 May;7(5):657-65.

Infectious Diseases: Viral Hepatitis

Enveloped viruses distinct from HBV induce dissemination of hepatitis D virus in vivo. – Perez-Vargas J, Amirache F, Boson B, Mialon C, Freitas N, Sureau C, Fusil F, Cosset FL. Nat Commun. 2019 May 8;10(1):2098.
In vivo combination of human anti-envelope glycoprotein E2 and -Claudin-1 monoclonal antibodies for prevention of hepatitis C virus infection. – Mailly L, Wrensch F, Heydmann L, Fauvelle C, Brignon N, Zeisel MB, Pessaux P, Keck ZY, Schuster C, Fuerst TR, Foung SKH, Baumert TF. Antiviral Res. 2019 Feb;162:136-14.
In Vivo Models of HDV Infection: Is Humanizing NTCP Enough? Giersch K, Dandri M. Viruses. 2021 Mar 31;13(4):588.
Clinical implications on HBV preS/S mutations and the effects of preS2 deletion on mitochondria, liver fibrosis, and cancer development. Liang YJ, Teng W, Chen CL, Sun CP, Teng RD, Huang YH, Liang KH, Chen YW, Lin CC, Su CW, Tao MH, Wu JC. Hepatology. 2021 Mar 5.
Optimization of AAV vectors to target persistent viral reservoirs. Colón-Thillet R, Jerome KR, Stone D. Virol J. 2021 Apr 23;18(1):85.
Animal models for the study of human hepatitis B and D virus infection: New insights and progress. Burwitz BJ, Zhou Z, Li W. Antiviral Res. 2020 Oct;182:104898.
MHC-E-Restricted CD8 + T Cells Target Hepatitis B Virus-Infected Human Hepatocytes. Burwitz BJ, Hashiguchi PK, Mansouri M, Meyer C, Gilbride RM, Biswas S, Womack JL, Reed JS, Wu HL, Axthelm MK, Hansen SG, Picker LJ, Früh K, Sacha JB. J Immunol. 2020 Apr 15;204(8):2169-2176.
Establishment of Humanized Mice for the Study of HBV. Lai F, Wee CYY, Chen Q. Front Immunol. 2021 Feb 19;12:638447.
Pharmacological Induction of a Progenitor State for the Efficient Expansion of Primary Human Hepatocytes. Unzu C, Planet E, Brandenberg N, Fusil F, Cassano M, Perez-Vargas J, Friedli M, Cosset FL, Lutolf MP, Wildhaber BE, Trono D. Hepatology. 2019 May;69(5):2214-2231.
Expansion, in vivo-ex vivo cycling, and genetic manipulation of primary human hepatocytes. Michailidis E, Vercauteren K, Mancio-Silva L, Andrus L, Jahan C, Ricardo-Lax I, Zou C, Kabbani M, Park P, Quirk C, Pyrgaki C, Razooky B, Verhoye L, Zoluthkin I, Lu WY, Forbes SJ, Chiriboga L, Theise ND, Herzog RW, Suemizu H, Schneider WM, Shlomai A, Meuleman P, Bhatia SN, Rice CM, de Jong YP. Proc Natl Acad Sci U S A. 2020 Jan 21;117(3):1678-1688.
Small Animal Models for Human Immunodeficiency Virus (HIV), Hepatitis B, and Tuberculosis: Proceedings of an NIAID Workshop. Akkina R, Barber DL, Bility MT, Bissig KD, Burwitz BJ, Eichelberg K, Endsley JJ, Garcia JV, Hafner R, Karakousis PC, Korba BE, Koshy R, Lambros C, Menne S, Nuermberger EL, Ploss A, Podell BK, Poluektova LY, Sanders-Beer BE, Subbian S, Wahl A. Curr HIV Res. 2020;18(1):19-28.
Woolly Monkey-HBV Infection in Squirrel Monkeys as a Surrogate Nonhuman Primate Model of HBV Infection. Chen CY, Winer BY, Chavez D, Guerra B, Brasky KM, Eng S, Salas E, Tam D, Simmons JH, Abee CR, Delaney WE, Ploss A, Lanford RE, Voitenleitner C. Hepatol Commun. 2020 Jan 3;4(3):371-386.
Animal Models of Hepatitis C Virus Infection. Ploss A, Kapoor A. Cold Spring Harb Perspect Med. 2020. 2020 May 1;10(5):a036970.
Humanized Mouse Models for the Study of Hepatitis C and Host Interactions. – Yong KSM, Her Z, Chen Q. Cells. 2019 Jun 17;8(6). pii: E604.
Updates in Hepatitis E virus (HEV) field; lessons learned from human liver chimeric mice. Sayed IM, Meuleman P. Rev Med Virol. 2020 Mar;30(2):e2086.
Cell and Animal Models for Studying Hepatitis B Virus Infection and Drug Development. Hu J, Lin YY, Chen PJ, Watashi K, Wakita T. Gastroenterology. 2019 Jan;156(2):338-354.
Animal Models for Hepatitis E Virus. Corneillie L, Banda DH, Meuleman P. Viruses. 2019 Jun 18;11(6). pii: E564.
Animal Models Used in Hepatitis C Virus Research. Berggren KA, Suzuki S, Ploss A. Int J Mol Sci. 2020 May 29;21(11):3869.
Hepatitis B Virus HBx Protein Mediates the Degradation of Host Restriction Factors through the Cullin 4 DDB1 E3 Ubiquitin Ligase Complex. Minor MM, Hollinger FB, McNees AL, Jung SY, Jain A, Hyser JM, Bissig KD, Slagle BL. Cells. 2020 Mar 30;9(4):834.
Updates in Hepatitis E virus (HEV) field; lessons learned from human liver chimeric mice. Sayed IM, Meuleman P. Rev Med Virol. 2019 Dec 13:e2086.
Efficacy of hepatitis B virus ribonuclease H inhibitors, a new class of replication antagonists, in FRG human liver chimeric mice. – Long KR, Lomonosova E, Li Q, Ponzar NL, Villa JA, Touchette E, Rapp S, Liley RM, Murelli RP, Grigoryan A, Buller RM, Wilson L, Bial J, Sagartz JE, Tavis JE. Antiviral Res. 2018 Jan;149:41-47.
Human liver chimeric mice provide a model for hepatitis B and C virus infection and treatment. – Bissig KD, Wieland SF, Tran P, Isogawa M, Le TT, Chisari FV, Verma IM. J Clin Invest. 2010 Mar;120(3):924-30
HBV infection-induced liver cirrhosis development in dual-humanised mice with human bone mesenchymal stem cell transplantation . – Yuan L, Jiang J, Liu X, Zhang Y, Zhang L, Xin J, Wu K, Li X, Cao J, Guo X, Shi D, Li J, Jiang L, Sun S, Wang T, Hou W, Zhang T, Zhu H, Zhang J, Yuan Q, Cheng T, Li J, Xia N. Gut. 2019 Jan 30.
A Chimeric Humanized Mouse Model by Engrafting the Human Induced Pluripotent Stem Cell-Derived Hepatocyte-Like Cell for the Chronic Hepatitis B Virus Infection. – Yuan L, Liu X, Zhang L, Li X, Zhang Y, Wu K, Chen Y, Cao J, Hou W, Zhang J, Zhu H, Yuan Q, Tang Q, Cheng T, Xia N. Front Microbiol. 2018 May 8;9:908.
Animal Models to Study Hepatitis C Virus Infection. – Burm R, Collignon L, Mesalam AA, Meuleman P. Front Immunol. 2018 May 14;9:1032.
Epigallocatechin gallate inhibits hepatitis B virus infection in human liver chimeric mice. – Lai YH, Sun CP, Huang HC, Chen JC, Liu HK, Huang C. BMC Complement Altern Med. 2018 Sep 6;18(1):248.
In vivo model systems for hepatitis B virus research. – Ortega-Prieto AM, Cherry C, Gunn H, Dorner M. ACS Infect Dis. 2018 Dec 12.
CRISPR-Cas9 gene editing of hepatitis B virus in chronically infected humanized mice. Stone D, Long KR, Loprieno MA, De Silva Feelixge HS, Kenkel EJ, Liley RM, Rapp S, Roychoudhury P, Nguyen T, Stensland L, Colón-Thillet R, Klouser LM, Weber ND, Le C, Wagoner J, Goecker EA, Li AZ, Eichholz K, Corey L, Tyrrell DL, Greninger AL, Huang ML, Polyak SJ, Aubert M, Sagartz JE, Jerome KR. Mol Ther Methods Clin Dev. 2020 Nov 26;20:258-275.
Mouse models for hepatitis B virus research. – Hwang JR, Park SG. Lab Anim Res. 2018 Sep;34(3):85-91.
Optimized HepaRG is a suitable cell source to generate the human liver chimeric mouse model for the chronic hepatitis B virus infection. – Yuan L, Liu X, Zhang L, Zhang Y, Chen Y, Li X, Wu K, Cao J, Hou W, Que Y, Zhang J, Zhu H, Yuan Q, Tang Q, Cheng T, Xia N. Emerg Microbes Infect. 2018 Aug 10;7(1):144.
Analysis of Hepatitis C Virus Particle Heterogeneity in Immunodeficient Human Liver Chimeric fah-/- Mice. – Andreo U, de Jong YP, Scull MA, Xiao JW, Vercauteren K, Quirk C, Mommersteeg MC, Bergaya S, Menon A, Fisher EA, Rice CM. Cell Mol Gastroenterol Hepatol. 2017 Jul 19;4(3):405-417
Polo-like-kinase 1 is a proviral host factor for hepatitis B virus replication. – Diab A, Foca A, Fusil F, Lahlali T, Jalaguier P, Amirache F, N’Guyen L, Isorce N, Cosset FL, Zoulim F, Andrisani O, Durantel D. Hepatology. 2017 Dec;66(6):1750-1765.
in Fah-/-Rag2-/- mice after pharmacological immunosuppression. – He Z, Zhang H, Zhang X, Xie D, Chen Y, Wangensteen KJ, Ekker SC, Firpo M, Liu C, Xiang D, Zi X, Hui L, Yang G, Ding X, Hu Y, Wang X. Am J Pathol. 2010 Sep;177(3):1311-9.
Rapid reversal of innate immune dysregulation in blood of patients and livers of humanized mice with HCV following DAA therapy. – Burchill MA, Roby JA, Crochet N, Wind-Rotolo M, Stone AE, Edwards MG, Dran RJ, Kriss MS, Gale M Jr, Rosen HR. PLoS One. 2017 Oct 17;12(10):e0186213.
Transmission of hepatitis E virus infection to human-liver chimeric FRG mice using patient plasma. – Sayed IM, Foquet L, Verhoye L, Abravanel F, Farhoudi A, Leroux-Roels G, Izopet J, Meuleman P. Antiviral Res. 2017 Feb 21;141:150-154
Studying HBV Infection and Therapy in Immune-Deficient NOD-Rag1-/-IL2RgammaC-null (NRG) Fumarylacetoacetate Hydrolase (Fah) Knockout Mice Transplanted with Human Hepatocytes. – Li F, Nio K, Yasui F, Murphy CM, Su L. Methods Mol Biol. 2017;1540:267-276.
Human embryonic stem cell-derived hepatoblasts are an optimal lineage stage for hepatitis C virus infection. – Yan F, Wang Y, Zhang W, Chang M, He Z, Xu J, Shang C, Chen T, Liu J, Wang X, Pei X, Wang Y. Hepatology. 2017 Sep;66(3):717-735.
Generation of Human Liver Chimeric Mice for the Study of Human Hepatotropic Pathogens. – von Schaewen M, Hrebikova G, Ploss A. Methods Mol Biol. 2016;1438:79-101.
Humanized mouse models for human viral hepatitis and related liver diseases. – Ren X, Zhou X. Chinese Science Bulletin, Volume 64, Issue 30: 3070-3076(2019)
Humanized mice efficiently engrafted with fetal hepatoblasts and syngeneic immune cells develop human monocytes and NK cells. – Billerbeck E, Mommersteeg MC, Shlomai A, Xiao JW, Andrus L, Bhatta A, Vercauteren K, Michailidis E, Dorner M, Krishnan A, Charlton MR, Chiriboga L, Rice CM, de Jong YP. J Hepatol. 2016 Aug;65(2):334-43.
Humanized mice in infectious diseases. – Ernst W. Comp Immunol Microbiol Infect Dis. 2016 Dec;49:29-38.
Mouse Systems to Model Hepatitis C Virus Treatment and Associated Resistance. – Mesalam AA, Vercauteren K, Meuleman P. Viruses. 2016 Jun 22;8(6).
Combinatorial RNA interference therapy prevents selection of pre-existing HBV variants in human liver chimeric mice. – Shih YM, Sun CP, Chou HH, Wu TH, Chen CC, Wu PY, Enya Chen YC, Bissig KD, Tao MH. Sci Rep. 2015 Oct;5:15259
Animal models for the study of HCV. – Vercauteren K, de Jong YP, Meuleman P. Curr Opin Virol. 2015 Aug;13:67-74.
Combinitorial RNA interference therapy prevents selection of pre-existing HBV variants in human liver chimeric mice. – Shih YM, Sun CP, Chou HH, Wu TH, Chen CC, Wu PY, Enya Chen YC, Bissig KD, Tao MH. Sci Rep. 2015 Oct;5:15259
Functional and Biochemical Characterization of Hepatitis C Virus (HCV) Particles Produced in a Humanized Liver Mouse Model. Calattini S, Fusil F, Mancip J, Dao Thi VL, Granier C, Gadot N, Scoazec JY, Zeisel MB, Baumert TF, Lavillette D, Dreux M, Cosset FL. J Biol Chem. 2015 Sep 18;290(38):23173-87.
Hepatitis C virus infects rhesus macaque hepatocytes and simianized mice. – Scull MA, Shi C, de Jong YP, Gerold G, Ries M, von Schaewen M, Donovan BM, Labitt RN, Horwitz JA, Gaska JM, Hrebikova G, Xiao JW, Flatley B, Fung C, Chiriboga L, Walker CM, Evans DT, Rice CM, Ploss A. Hepatology. 2015 Jul;62(1):57-67.
HCV animal models and liver disease. – Vercauteren K, de Jong YP, Meuleman P. J Hepatol. 2014 Nov;61(1 Suppl):S26-33.
New generation humanized mice for virus research: Comparative aspects and future prospects. – Akkina R. Virology. 2013 Jan 5;435(1):14-28.
Study of hepatitis C virus entry in genetically humanized mice. – Dorner M, Rice CM, Ploss A. Methods. 2013 Feb;59(2):249-57.
A humanized mouse model to study hepatitis C virus infection, immune response, and liver disease. Washburn ML, Bility MT, Zhang L, Kovalev GI, Buntzman A, Frelinger JA, Barry W, Ploss A, Rice CM, Su L. Gastroenterology. 2011 Apr;140(4):1334-44.
Deconstructing hepatitis C virus infection in humanized mice. – Dorner M, Ploss A. Ann N Y Acad Sci. 2011 Dec;1245:59-62.
New horizons for studying human hepatotropic infections. de Jong YP, Rice CM, Ploss A. J Clin Invest. 2010 Mar;120(3):650-3.

NASH & NAFLD

Human hepatocyte PNPLA3 148M exacerbates rapid non-alcoholic steatohepatitis development in chimeric mice. Kabbani M, Michailidis E, Steensels S, Fulmer CG, Luna JM, Le Pen J, Tardelli M, Razooky B, Ricardo-Lax I, Zou C, Zeck B, Stenzel AF, Quirk C, Foquet L, Ashbrook AW, Schneider WM, Belkaya S, Lalazar G, Liang Y, Pittman M, Devisscher L, Suemizu H, Theise ND, Chiriboga L, Cohen DE, Copenhaver R, Grompe M, Meuleman P, Ersoy BA, Rice CM, de Jong YP. bioRxiv 2020.11.19.387613.
A human liver chimeric mouse model for non-alcoholic fatty liver disease. Bissig-Choisat B, Alves-Bezerra M, Zorman B, Ochsner SA, Barzi M, Legras X, Yang D, Borowiak M, Dean AM, York RB, Galvan NTN, Goss J, Lagor WR, Moore DD, Cohen DE, McKenna NJ, Sumazin P, Bissig KD. JHEP Reports, Volume 0, Issue 0, 100281.

Cardiovascular Disease, Hypercholesterolaemia, & Lipoprotein Metabolism

New insights from liver-humanized mice on cholesterol lipoprotein metabolism and LXR-agonist pharmacodynamics in humans. Minniti ME, Pedrelli M, Vedin LL, Delbès AS, Denis RGP, Öörni K, Sala C, Pirazzini C, Thiagarajan D, Nurmi HJ, Grompe M, Mills K, Garagnani P, Ellis ECS, Strom SC, Luquet SH, Wilson EM, Bial J, Steffensen KR, Parini P. Hepatology. 2019 Nov 30.
Lipoprotein(a) Cellular Uptake Ex Vivo and Hepatic Capture In Vivo Is Insensitive to PCSK9 Inhibition With Alirocumab. Chemello K, Beeské S, Trang Tran TT, Blanchard V, Villard EF, Poirier B, Le Bail JC, Dargazanli G, Ho-Van-Guimbal S, Boulay D, Bergis O, Pruniaux MP, Croyal M, Janiak P, Guillot E, Lambert G. JACC Basic Transl Sci. 2020 May 6;5(6):549-557.
Lipoprotein(a): An Enigmatic Sheep in the Lipoprotein Herd. Shapiro MD, Fazio S. JACC Basic Transl Sci. 2020;5(6):558-560. Published 2020 Jun 22.
Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model. – Bissig-Choisat B, Wang L, Legras X, Saha PK, Chen L, Bell P, Pankowicz FP, Hill MC, Barzi M, Kettlun-Leyton C, Leung HC, Kruse RL, Himes RW, Foss JA, Wilson JM, Chan L, Lagor WR, Bissig KD. Nat Commun. 2015 Jun 17;6:7339
Mice with chimeric livers are an improved model for human lipoprotein metabolism – Ellis EC, Nauglers S, Parini P, Mörk LM, Jorns C, Zemack H, Sandblom AL, Björkhem I, Ericzon BG, Wilson EM, Strom SC, Grompe M. PLoS One. 2013 Nov 4;8(11)
A drug screen using human iPSC-derived hepatocyte-like cells reveals cardiac glycosides as a potential treatment for hypercholesterolemia. – Cayo MA, Mallanna SK, Di Furio F, Jing R, Tolliver LB, Bures M, Urick A, Noto FK, Pashos EE, Greseth MD, Czarnecki M, Traktman P, Yang W, Morrisey EE, Grompe M, Rader DJ, Duncan SA. Cell Stem Cell. 2017 Apr 6;20(4):478-489

Urea Cycle & Metabolic Disorders

A liver-humanized mouse model of carbamoyl phosphate synthetase 1-deficiency. Srinivasan RC, Zabulica M, Hammarstedt C, Wu T, Gramignoli R, Kannisto K, Ellis E, Karadagi A, Fingerhut R, Allegri G, Rüfenacht V, Thöny B, Häberle J, Nuoffer JM, Strom SC. J Inherit Metab Dis. 2019 Mar 6.
Correction of a urea cycle defect after ex vivo gene editing of human hepatocytes. Zabulica M, Srinivasan RC, Akcakaya P, Allegri G, Bestas B, Firth M, Hammarstedt C, Jakobsson T, Jakobsson T, Ellis E, Jorns C, Makris G, Scherer T, Rimann N, van Zuydam NR, Gramignoli R, Forslöw A, Engberg S, Maresca M, Rooyackers O, Thöny B, Häberle J, Rosen B, Strom SC. Mol Ther. 2021 Jan 21:S1525-0016(21)00024-1.
Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes. Ginn SA, Amaya AK, Liao SHY, Zhu E, Cunningham SC, Lee M, Hallwirth CV, Logan GJ, Tay SS, Cesare AJ, Pickett HA, Grompe M, Dilworth K, Lisowski L, Alexander IE. JHEP Reports, Volume 0, Issue 0, 100065.
Human hepatocyte transplantation corrects the inherited metabolic liver disorder arginase deficiency in mice. – Angarita SAK, Truong B, Khoja S, Nitzahn M, Rajbhandari AK, Zhuravka I, Duarte S, Lin MG, Lam AK, Cederbaum SD, Lipshutz GS. Mol Genet Metab. 2018 Jun;124(2):114-123.
New potential cell source for hepatocyte transplantation: discard livers from metabolic disease liver transplants. – Gramignoli R, Tahan V, Dorko K, Skvorak KJ, Hansel MC, Zhao W, Venkataramanan R, Ellis EC, Jorns C, Ericzon BG, Rosenborg S, Kuiper R, Soltys KA, Mazariegos GV, Fox IJ, Wilson EM, Grompe M, Strom SC. Stem Cell Res. 2013 Jul;11(1):563-73
CPS-1 Humanized Liver in FRGN Mouse as Model for Genetic Disorder. Srinivasan R, Zabulica M, Hammerstadt C, Gramignoli R, Strom S. Transplantation 103:S21. 2019 Sep.

Drug Metabolism & Toxicology

Fibroblast Growth Factor Signaling Controls Liver Size in Mice with Humanized Livers. – Naugler WE, Tarlow BD, Fedorov LM, Taylor M, Pelz C, Li B, Darnell J, Grompe M. Gastroenterology. 2015 Sep;149(3):728-40.
Endoplasmic Reticulum Stress-Induced Upregulation of STARD1 Promotes Acetaminophen-Induced Acute Liver Failure. Torres S, Baulies A, Insausti-Urkia N, Alarcón-Vila C, Fucho R, Solsona-Vilarrasa E, Núñez S, Robles D, Ribas V, Wakefield L, Grompe M, Lucena MI, Andrade RJ, Win S, Aung TA, Kaplowitz N, García-Ruiz C, Fernández-Checa JC. Gastroenterology. 2019 Aug;157(2):552-568.CYP-humanized and Human Liver Chimeric Mouse Models for Studying Xenobiotic Metabolism and Toxicity. Bissig KD, Han W, Barzi M, Kovalchuk N, Ding L, Fan X, Pankowicz F, Zhang QY, Ding X Drug Metabolism and Disposition August 9, 2018.
The pharmacokinetics and metabolism of diclofenac in chimeric humanized and murinized FRG mice. – Wilson CE, Dickie AP, Schreiter K, Wehr R, Wilson EM, Bial J, Scheer N, Wilson ID, Riley RJ. Arch Toxicol. 2018 Jun;92(6):1953-1967.
Chimeric mice with humanized liver: tools for the study of drug metabolism, excretion, and toxicity. – Strom SC, Davila J, Grompe M. Methods Mol Biol. 2010;640:491-509
A novel humanized mouse lacking murine P450 oxidoreductase for studying human drug metabolism. – Barzi M, Pankowicz FP, Zorman B, Liu X, Legras X, Yang D, Borowiak M, Bissig-Choisat B, Sumazin P, Li F, Bissig KD. Nat Commun. 2017 Jun 28;8(1):39.
Disrupted Murine Gut-to-Human Liver Signaling Alters Bile Acid Homeostasis in Humanized Mouse Liver Models. – Chow EC, Quach HP, Zhang Y, Wang JZ, Evans DC, Li AP, Silva J, Tirona RG, Lai Y, Pang KS. J Pharmacol Exp Ther. 2017 Jan;360(1):174-191.
The pharmacokinetics and metabolism of lumiracoxib in chimeric humanized and murinized FRG mice. – Dickie AP, Wilson CE, Schreiter K, Wehr R, Wilson EM, Bial J, Scheer N, Wilson ID, Riley RJ. Biochem Pharmacol. 2017 Jul 1;135-150
A comparison between genetically humanized and chimeric liver humanized mouse models for studies in drug metabolism and toxicity. – Scheer N, Wilson ID. Drug Discov Today. 2016 Feb;21(2):250-63.
Functional Integrity of Chimeric (Humanized) Mouse Liver: Enzyme Zonation, Physiologic Spaces, and Hepatic Enzymes and Transporters. – Chow EC, Wang JZ, Quach HP, Tang H, Evans DC, Li AP, Silva J, Pang KS. Drug Metab Dispos. 2016 Sep;44(9):1524-35.
LC-ESI-MS/MS quantification of 4β-hydroxycholesterol and cholesterol in plasma samples of limited volume. – Xu Y, Yuan Y, Smith L, Edom R, Weng N, Mamidi R, Silva J, Evans DC, Lim HK. J Pharm Biomed Anal. 2013 Nov;85:145-54.
Troglitazone metabolism and transporter effects in chimeric mice: a comparison between chimeric humanized and chimeric murinized FRG mice. – Samuelsson K, Pickup K, Sarda S, Foster JR, Randall K, Abrahamsson A, Jacobsen M, Weidolf L, Wilson I. Xenobiotica. 2014 Jan;44(2):186-95
Response to “Can ‘humanized’ mice improve drug development in the 21st century?” – Bissig KD, Grompe M. Trends Pharmacol Sci. 2013 Aug;34(8):425
In vitro inhibition and induction of human liver cytochrome P450 enzymes by NTBC and its metabolism in human liver microsomes. – Neat JN, Wolff A, Kazmi F, Prentiss P, Buckley D, Wilson EM, Bial J, Cedo M. Bagi, Grompe M, Parkinson A. August 2010 Drug Metabolism Reviews

Regenerative Medicine

In Vivo Lineage Tracing of Polyploid Hepatocytes Reveals Extensive Proliferation during Liver Regeneration. Matsumoto T, Wakefield L, Tarlow BD, Grompe M. Cell Stem Cell. 2019 Dec 11. pii: S1934-5909(19)30469-2.
Synthetic Maturation of Multilineage Human Liver Organoids via Genetically Guided Engineering. Velazquez J, Ryan LeGraw, Farzaneh Moghadam, Yuqi Tan, Jacquelyn Kilbourne, Joshua Hislop, S Liu, Davy Cats, Susana M. Chuva de Sousa Lopes, Christopher Plaisier, Patrick Cahan, Samira Kiani, Ebrahimkhani MR.
Absence of Atg7 in the liver disturbed hepatic regeneration after liver injury. Römermann D, Ansari N, Schultz-Moreira AR, Michael A, Marhenke S, Hardtke-Wolenski M, Longerich T, Manns MP, Wedemeyer H, Vogel A, Buitrago-Molina LE. Liver Int. 2020 Mar 6.
Chimeric liver transplantation reveals interspecific graft remodelling. – Oldani G, Peloso A, Vijgen S, Wilson EM, Slits F, Gex Q, Morel P, Delaune V, Orci LA, Yamaguchi T, Kobayashi T, Rubbia-Brandt L, Nakauchi H, Lacotte S, Toso C. J Hepatol. 2018 Nov;69(5):1025-1036.
Inflammatory Cytokine TNFα Promotes the Long-Term Expansion of Primary Hepatocytes in 3D Culture. – Peng WC, Logan CY, Fish M, Anbarchian T, Aguisanda F, Álvarez-Varela A, Wu P, Jin Y, Zhu J, Li B, Grompe M, Wang B, Nusse R. Cell. 2018 Nov 29;175(6):1607-1619.e15.
Adult Mouse Liver Contains Two Distinct Populations of Cholangiocytes. – Li B, Dorrell C, Canaday PS, Pelz C, Haft A, Finegold M, Grompe M. Stem Cell Reports. 2017 Aug 8;9(2):478-489.
Agonist c-Met Monoclonal Antibody Augments the Proliferation of hiPSC-derived Hepatocyte-Like Cells and Improves Cell Transplantation Therapy for Liver Failure in Mice. Yuan L, Zhang Y, Liu X, Chen Y, Zhang L, Cao J, Li X, Wang M, Wu K, Zhang J, Liu G, Tang Q, Yuan Q, Cheng T, Xia N. Theranostics. 2019 Apr 6;9(7):2115-2128.
Bioengineering considerations in liver regenerative medicine. – Ogechi Ogoke, Janet Oluwole, Natesh Parashurama. J Biol Eng. 2017; 11: 46.
In situ expansion of engineered human liver tissue in a mouse model of chronic liver disease. – Stevens KR, Scull MA, Ramanan V, Fortin CL, Chaturvedi RR, Knouse KA, Xiao JW, Fung C, Mirabella T, Chen AX, McCue MG, Yang MT, Fleming HE, Chung K, de Jong YP, Chen CS, Rice CM, Bhatia SN. Sci Transl Med. 2017 Jul 19;9(399).
Towards a Humanized Mouse Model of Liver Stage Malaria Using Ectopic Artificial Livers. – Ng S, March S, Galstian A, Gural N, Stevens KR, Mota MM, Bhatia SN. Sci Rep. 2017 Mar 31;7:45424.
Humanization of immunodeficient animals for the modeling of transplantation, graft versus host disease and regenerative medicine Adigbli G, Ménoret S, Cross AR, Hester J, Issa F, Anegon I. Transplantation. 2020 Feb 14.
Fibroblast Growth Factor Signaling Controls Liver Size in Mice with Humanized Livers. – Naugler WE, Tarlow BD, Fedorov LM, Taylor M, Pelz C, Li B, Darnell J, Grompe M. Gastroenterology. 2015 Sep;149(3):728-40.
Natural Killer Cells-Produced IFN-γ Improves Bone Marrow-Derived Hepatocytes Regeneration in Murine Liver Failure Model. – Li L, Zeng Z, Qi Z, Wang X, Gao X, Wei H, Sun R, Tian Z. Sci Rep. 2015 Sep 8;5:13687.
The organoid-initiating cells in mouse pancreas and liver are phenotypically and functionally similar. – Dorrell C, Tarlow B, Wang Y, Canaday PS, Haft A, Schug J, Streeter PR, Finegold MJ, Shenje LT, Kaestner KH, Grompe M. Stem Cell Res. 2014 Sep;13(2):275-83.
Bipotential adult liver progenitors are derived from chronically injured mature hepatocytes. – Tarlow BD, Pelz C, Naugler WE, Wakefield L, Wilson EM, Finegold MJ, Grompe M. Cell Stem Cell. 2014 Nov 6;15(5):605-18
Mouse liver repopulation with hepatocytes generated from human fibroblasts. – Zhu S, Rezvani M, Harbell J, Mattis AN, Wolfe AR, Benet LZ, Willenbring H, Ding S. Nature. 2014 Apr 3;508(7494):93-7.
In vitro expansion of single Lgr5+ liver stem cells induced by Wnt-driven regeneration. – Huch M, Dorrell C, Boj SF, van Es JH, Li VS, van de Wetering M, Sato T, Hamer K, Sasaki N, Finegold MJ, Haft A, Vries RG, Grompe M, Clevers H. Nature. 2013 Feb 14;494(7436):247-5
Frequent aneuploidy among normal human hepatocytes. – Duncan AW, Hanlon Newell AE, Smith L, Wilson EM, Olson SB, Thayer MJ, Strom SC, Grompe M. Gastroenterology. 2012 Jan;142(1):25-8.
Therapeutic liver reconstitution with murine cells isolated long after death. – Erker L, Azuma H, Lee AY, Guo C, Orloff S, Eaton L, Benedetti E, Jensen B, Finegold M, Willenbring H, Grompe M. Gastroenterology. 2010 Sep;139(3):1019-29
Repopulation of adult and neonatal mice with human hepatocytes: a chimeric animal model. – Bissig KD, Le TT, Woods NB, Verma IM. Proc Natl Acad Sci U S A. 2007 Dec 18;104(51):20507-11

Stem Cells & Organoids

Long-Term Expansion of Functional Mouse and Human Hepatocytes as 3D Organoids. – Hu H, Gehart H, Artegiani B, LÖpez-Iglesias C, Dekkers F, Basak O, van Es J, Chuva de Sousa Lopes SM, Begthel H, Korving J, van den Born M, Zou C, Quirk C, Chiriboga L, Rice CM, Ma S, Rios A, Peters PJ, de Jong YP, Clevers H. Cell. 2018 Nov 29;175(6):1591-1606.e19.
Liver sinusoidal endothelial cells promote the differentiation and survival of mouse vascularised hepatobiliary organoids. Yap KK, Gerrand YW, Dingle AM, Yeoh GC, Morrison WA, Mitchell GM. Biomaterials. 2020 Aug;251:120091.
A Roadmap for Human Liver Differentiation from Pluripotent Stem Cells. – Ang LT, Tan AKY, Autio MI, Goh SH, Choo SH, Lee KL, Tan J, Pan B, Lee JJH, Lum JJ, Lim CYY, Yeo IKX, Wong CJY, Liu M, Oh JLL, Chia CPL, Loh CH, Chen A, Chen Q, Weissman IL, Loh KM, Lim B. Cell Rep. 2018 Feb 20;22(8):2190-2205.
Human ESC-derived expandable hepatic organoids enable therapeutic liver repopulation and pathophysiological modeling of alcoholic liver injury. Wang S, Wang X, Tan Z, Su Y, Liu J, Chang M, Yan F, Chen J, Chen T, Li C, Hu J, Wang Y. Cell Res. 2019 Oct 18.
Expandable liver organoids generated from human chemically derived hepatic progenitor enable alcoholic liver modeling. Y Kim, S Yoon, MH Kim, DH Hong, D Choi. Korean J Transplant 2020; 34(Suppl 1): S126-S126.
Large-scale Production of LGR5-positive Bipotential Human Liver Stem Cells. Schneeberger K, Sánchez-Romero N, Ye S, van Steenbeek FG, Oosterhoff LA, Pla Palacin I, Chen C, van Wolferen ME, van Tienderen G, Lieshout R, Colemonts-Vroninks H, Schene I, Hoekstra R, Verstegen MMA, van der Laan LJW, Penning LC, Fuchs SA, Clevers H, De Kock J, Baptista PM, Spee B. Hepatology. 2019 Nov 12.
Alternative Cell Sources for Liver Parenchyma Repopulation: Where Do We Stand? Tricot T, De Boeck J, Verfaillie C. Cells. 2020 Feb 28;9(3). pii: E566.
A small molecule Hedgehog agonist HhAg1.5 mediated reprogramming breaks the quiescence of noninjured liver stem cells for rescuing liver failure. – Mitra A, Yan J, Zhang L, Li S. Transl Res. 2018 Oct 19.
Transplantable Liver Organoids, Too Many Cell Types to Choose: a Need for Scientific Self-Organization. Baptista, PM. Curr Transpl Rep 7, 18–23 (2020).
A Chimeric Humanized Mouse Model by Engrafting the Human Induced Pluripotent Stem Cell-Derived Hepatocyte-Like Cell for the Chronic Hepatitis B Virus Infection. – Yuan L, Liu X, Zhang L, Li X, Zhang Y, Wu K, Chen Y, Cao J, Hou W, Zhang J, Zhu H, Yuan Q, Tang Q, Cheng T, Xia N. Front Microbiol. 2018 May 8;9:908.
Expansion and differentiation of human hepatocyte-derived liver progenitor-like cells and their use for the study of hepatotropic pathogens. Fu GB, Huang WJ, Zeng M, Zhou X, Wu HP, Liu CC, Wu H, Weng J, Zhang HD, Cai YC, Ashton C, Ding M, Tang D, Zhang BH, Gao Y, Yu WF, Zhai B, He ZY, Wang HY, Yan HX. Cell Res. 2019 Jan;29(1):8-22.
Optimized HepaRG is a suitable cell source to generate the human liver chimeric mouse model for the chronic hepatitis B virus infection. – Yuan L, Liu X, Zhang L, Zhang Y, Chen Y, Li X, Wu K, Cao J, Hou W, Que Y, Zhang J, Zhu H, Yuan Q, Tang Q, Cheng T, Xia N. Emerg Microbes Infect. 2018 Aug 10;7(1):144.
Biofabrication of Autologous Human Hepatocytes for Transplantation: How Do We Get There? Agarwal N, Popovic B, Martucci NJ, Fraunhoffer NA, Soto-Gutierrez A. Gene Expr. 2019 Apr 18;19(2):89-95.
Three-dimensional bioprinted hepatorganoids prolong survival of mice with liver failure. Yang H, Sun L, Pang Y, Hu D, Xu H, Mao S, Peng W, Wang Y, Xu Y, Zheng YC, Du S, Zhao H, Chi T, Lu X, Sang X, Zhong S, Wang X, Zhang H, Huang P, Sun W, Mao Y. Gut. 2021 Mar;70(3):567-574.
Bipotential adult liver progenitors are derived from chronically injured mature hepatocytes. – Tarlow BD, Pelz C, Naugler WE, Wakefield L, Wilson EM, Finegold MJ, Grompe M. Cell Stem Cell. 2014 Nov 6;15(5):605-18
Human embryonic stem cell-derived hepatoblasts are an optimal lineage stage for hepatitis C virus infection. – Yan F, Wang Y, Zhang W, Chang M, He Z, Xu J, Shang C, Chen T, Liu J, Wang X, Pei X, Wang Y. Hepatology. 2017 Sep;66(3):717-735.
Conversion of Human Gastric Epithelial Cells to Multipotent Endodermal Progenitors using Defined Small Molecules. – Wang Y, Qin J, Wang S, Zhang W, Duan J, Zhang J, Wang X, Yan F, Chang M, Liu X, Feng B, Liu J, Pei X. Cell Stem Cell. 2016 Oct 6;19(4):449-461.
Generation of functional human hepatocytes in vitro: current status and future prospects. Yamaguchi T, Matsuzaki J, Katsuda T, Saito Y, Saito H, Ochiya T. Inflamm Regen. 2019 Jul 2;39:13.
The Use of Induced Pluripotent Stem Cells for the Study and Treatment of Liver Diseases. – Hansel MC, Davila JC, Vosough M, Gramignoli R, Skvorak KJ, Dorko K, Marongiu F, Blake W, Strom SC. Curr Protoc Toxicol. 2016 Feb 1;67:14.13.1-14.13.27.
Generation of integration-free induced hepatocyte-like cells from mouse fibroblasts. – Kim J, Kim KP, Lim KT, Lee SC, Yoon J, Song G, Hwang SI, Schöler HR, Cantz T, Han DW. Sci Rep. 2015 Oct 27;5:15706.
MicroRNA-199a-5p inhibition enhances the liver repopulation ability of human embryonic stem cell-derived hepatic cells. – Möbus S, Yang D, Yuan Q, Lüdtke TH, Balakrishnan A, Sgodda M, Rani B, Kispert A, Araúzo-Bravo MJ, Vogel A, Manns MP, Ott M, Cantz T, Sharma AD. J Hepatol. 2015 Jan;62(1):101-10.
Stem cell differentiation and human liver disease. – Zhou WL, Medine CN, Zhu L, Hay DC. World J Gastroenterol. 2012 May 7;18(17):2018-25.
Stem cells and liver regeneration. – Duncan AW, Dorrell C, Grompe M. Gastroenterology. 2009 Aug;137(2):466-81.

Cancer Biology

A human liver cell atlas reveals heterogeneity and epithelial progenitors. Aizarani N, Saviano A, Sagar, Mailly L, Durand S, Herman JS, Pessaux P, Baumert TF, Grün D. Nature. 2019 Aug; 572(7768):199-204.
Innovations, challenges, and minimal information for standardization of humanized mice. Stripecke R, Münz C, Schuringa JJ, Bissig KD, Soper B, Meeham T, Yao LC, Di Santo JP, Brehm M, Rodriguez E, Wege AK, Bonnet D, Guionaud S, Howard KE, Kitchen S, Klein F, Saeb-Parsy K, Sam J, Sharma AD, Trumpp A, Trusolino L, Bult C, Shultz L. EMBO Mol Med. 2020 Jul 7;12(7):e8662.
Mouse models of oncoimmunology in hepatocellular carcinoma. E Bresnahan, KE Lindblad, M Ruiz de Galarreta, A Lujambio. Clin Cancer Res. 2020 Apr 23:clincanres.2923.2020.
Uncovering biological factors that regulate hepatocellular carcinoma growth using patient derived xenograft assays. Zhu M, Li L, Lu T, Yoo H, Zhu J, Gopal P, Wang SC, Porempka MR, Rich NE, Kagan S, Odewole M, Renteria V, Waljee AK, Wang T, Singal AG, Yopp AC, Zhu H. Hepatology. 2020 Jan 3.
Doublecortin-like Kinase 1 Promotes Hepatocyte Clonogenicity and Oncogenic Programming via Non-Canonical β-catenin-dependent Mechanism. Ali N, Nguyen CB, Chandrakesan P, Wolf RF, Qu D, May R, Goretsky T, Fazili J, Barrett TA, Li M, Huycke MM, Bronze MS, Houchen CW. Sci Rep. 2020 Jun 29;10(1):10578.
Novel patient-derived preclinical models of liver cancer. Bresnahan E, Ramadori P, Heikenwalder M, Zender L, Lujambio A. J Hepatol. 2020 Feb;72(2):239-249.
The breakthrough in primary human hepatocytes in vitro expansion. Feng M, Kong R, Pan Y. Cancer Biol Med. 2019 Feb;16(1):1-3.
Knockdown of Anillin Actin Binding Protein Blocks Cytokinesis in Hepatocytes and Reduces Liver Tumor Development in Mice Without Affecting Regeneration . – Zhang S, Nguyen LH, Zhou K, Tu HC, Sehgal A, Nassour I, Li L, Gopal P, Goodman J, Singal AG, Yopp A, Zhang Y, Siegwart DJ, Zhu H. Gastroenterology. 2018 Apr;154(5):1421-1434.
Mouse Models for Immunotherapy in Hepatocellular Carcinoma. E Li, L Lin, CW Chen, DL Ou. Cancers (Basel). 2019 Nov 15;11(11):1800.

Murine model of hepatic breast cancer. – Rikhi R, Wilson EM, Deas O, Svalina MN, Bial J, Mansoor A, Cairo S, Keller C. Biochem Biophys Rep. 2016 Aug 4;8:1-5.
Novel patient-derived xenograft and cell line models for therapeutic testing of pediatric liver cancer. – Bissig-Choisat B, Kettlun-Leyton C, Legras XD, Zorman B, Barzi M, Chen LL, Amin MD, Hyuang YH, Pautler RG, Hampton OA, Prakash MM, Yang D, Borowiak M, Muzny D, Doddapaneni HV, Hu J, Shi Y, Gaber MW, Hicks MJ, Thompson PA, Lu Y, Mills GB, Finegold M, Goss JA, Parsons DW, Vasudevan SA, Sumazin P, Lopez-Terrada D, Bissig KD. J Hepatol. 2016 Aug;65(2):325-33

Other Infectious Diseases

Alcohol-and-HIV-Induced Lysosomal Dysfunction Regulates Extracellular Vesicles Secretion in Vitro and in Liver-Humanized Mice. Dagur RS, New-Aaron M, Ganesan M, Wang W, Romanova S, Kidambi S, Kharbanda KK, Poluektova LY, Osna NA. Biology (Basel). 2021 Jan 5;10(1):29.
Consumptive coagulopathy of severe yellow fever occurs independently of hepatocellular tropism and massive hepatic injury. Bailey AL, Kang LI, de Assis Barros D’Elia Zanella LGF, Silveira CGT, Ho YL, Foquet L, Bial G, McCune BT, Duarte-Neto AN, Thomas A, Raué HP, Byrnes K, Kallas EG, Slifka MK, Diamond MS. Proc Natl Acad Sci U S A. 2020 Dec 22;117(51):32648-32656.

In Vitro

In Vitro Expansion of Primary Human Hepatocytes with Efficient Liver Repopulation Capacity.. – Zhang K, Zhang L, Liu W, Ma X, Cen J, Sun Z, Wang C, Feng S, Zhang Z, Yue L, Sun L, Zhu Z, Chen X, Feng A, Wu J, Jiang Z, Li P, Cheng X, Gao D, Peng L, Hui L. Cell Stem Cell. 2018 Dec 6;23(6):806-819.e4.
Microphysical space of a liver sinusoid decice enables simplified long-term maintenance of chimeric mouse-expanded human hepatocytes. – Maher SP, Crouse RB, Conway AJ, Bannister EC, Achyuta AK, Clark AY, Sinatra FL, Cuiffi JD, Adams JH, Kyle DE, Saadi WM. Biomed Microdevices. 2014 Octl 16(5):727-36
Robust expansion of human hepatocytes in Fah-/- / Rag2-/- / Il2rg-/- mice. – Azuma H, Paulk N, Ranade A, Dorrell C, Al-Dhalimy M, Ellis E, Strom S, Kay MA, Finegold M, Grompe M. Nat Biotechnol. 2007 Aug;25(8):903-10.

Pig & Rat Models

Curative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1. – Hickey RD, Mao SA, Glorioso J, Elgilani F, Amiot B, Chen H, Rinaldo P, Marler R, Jiang H, DeGrado TR, Suksanpaisan L, O’Connor MK, Freeman BL, Ibrahim SH, Peng KW, Harding CO, Ho CS, Grompe M, Ikeda Y, Lillegard JB, Russell SJ, Nyberg SL. Sci Transl Med. 2016 Jul 27;8(349):349ra99.
Generation and characterization of rat liver stem cell lines and their engraftment in a rat model of liver failure. – Kuijk EW, Rasmussen S, Blokzijl F, Huch M, Gehart H, Toonen P, Begthel H, Clevers H, Geurts AM, Cuppen E. Sci Rep. 2016 Feb 26;6:22154.
Fumarylacetoacetate hydrolase deficient pigs are a novel large animal model of metabolic liver disease. – Hickey RD, Mao SA, Glorioso J, Lillegard JB, Fisher JE, Amiot B, Rinaldo P, Harding CO, Marler R, Finegold MJ, Grompe M, Nyberg SL. Stem Cell Res. 2014 Jul;13(1):144-53.
Autologous Gene and Cell Therapy Provides Safe and Long-Term Curative Therapy in A Large Pig Model of Hereditary Tyrosinemia Type 1. Raymond D. Hickey, Clara T. Nicolas, Kari Allen, Shennen Mao, Faysal Elgilani, Jaime Glorioso, Bruce Amiot, Caitlin VanLith, Rebekah Guthman, Zeji Du, Harvey Chen, Cary O. Harding, Robert A. Kaiser, Scott L. Nyberg, Joseph B. Lillegard. Cell Transplant. 2019 Jan; 28(1): 79–88. Published online 2018.