Adeno-Associated Virus Gene Therapy for Liver Disease

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) · Nature Medicine · doi:10.1038/nm1358 · Cited by 2,163 · see citing works

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

Nathwani, A. C., Tuddenham, E. G. D., Rangarajan, S., Rosales, C., McIntosh, J., Linch, D. C., Chowdary, P., Riddell, A., Pie, A. J., Harrington, C., et al. (2011) · New England Journal of Medicine · doi:10.1056/nejmoa1108046 · Cited by 1,864 · see citing works

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

Gao, G.-P., Alvira, M. R., Wang, L., Calcedo, R., Johnston, J., & Wilson, J. M. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.182412299 · Cited by 1,520 · see citing works

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

Zincarelli, C., Soltys, S., Rengo, G., & Rabinowitz, J. E. (2008) · Molecular Therapy · doi:10.1038/mt.2008.76 · Cited by 1,490 · see citing works

Principles of Anatomy and Physiology

Baker, G. F., Tortora, G. J., & Nostakos, N. P. A. (1976) · AJN American Journal of Nursing · doi:10.2307/3423898 · Cited by 1,322 · see citing works

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

Nathwani, A. C., Reiss, U. M., Tuddenham, E. G. D., Rosales, C., Chowdary, P., McIntosh, J., Della Peruta, M., Lheriteau, E., Patel, N., Raj, D., et al. (2014) · New England Journal of Medicine · doi:10.1056/nejmoa1407309 · Cited by 1,273 · see citing works

Gene therapy returns to centre stage

Naldini, L. (2015) · Nature · doi:10.1038/nature15818 · Cited by 1,184 · cited 1× · see citing works

Clades of Adeno-Associated Viruses Are Widely Disseminated in Human Tissues

Gao, G., Vandenberghe, L. H., Alvira, M. R., Lu, Y., Calcedo, R., Zhou, X., & Wilson, J. M. (2004) · Journal of Virology · doi:10.1128/jvi.78.12.6381-6388.2004 · Cited by 1,070 · see citing works

Effect of genome size on AAV vector packaging

Wu, Z., Yang, H., & Colosi, P. (2010) · Molecular Therapy · doi:10.1038/mt.2009.255 · Cited by 1,025 · see citing works

Treatment of Leber Congenital Amaurosis Due to <i>RPE65</i> Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

Hauswirth, W. W., Aleman, T. S., Kaushal, S., Cideciyan, A. V., Schwartz, S. B., Wang, L., Conlon, T. J., Boye, S. L., Flotte, T. R., Byrne, B. J., et al. (2008) · Human Gene Therapy · doi:10.1089/hum.2008.107 · Cited by 954 · see citing works

Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses

Calcedo, R., Vandenberghe, L. H., Gao, G., Lin, J., & Wilson, J. M. (2009) · The Journal of Infectious Diseases · doi:10.1086/595830 · Cited by 808 · see citing works

A morphologic study of deoxyribonucleic acid synthesis and cell proliferation in regenerating rat liver; autoradiography with thymidine-H3.

Grisham, J. W. (1962) · PubMed · Cited by 772 · see citing works

Efficient presentation of exogenous antigen by liver endothelial cells to CD8+ T cells results in antigen-specific T-cell tolerance

Limmer, A., Ohl, J., Kurts, C., Ljunggren, H.-G., Reiss, Y., Groettrup, M., Momburg, F., Arnold, B., & Knolle, P. A. (2000) · Nature Medicine · doi:10.1038/82161 · Cited by 758 · see citing works

In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses

Grimm, D., Lee, J. S., Wang, L., Desai, T., Akache, B., Storm, T. A., & Kay, M. A. (2008) · Journal of Virology · doi:10.1128/jvi.00254-08 · Cited by 696 · see citing works

AAV vector integration sites in mouse hepatocellular carcinoma

Donsante, A., Miller, D. G., Li, Y., Vogler, C., Brunt, E. M., Russell, D. W., & Sands, M. S. (2007) · Science · doi:10.1126/science.1142658 · Cited by 662 · see citing works

Hepatic T cells and liver tolerance

Crispe, I. N. (2003) · Nature Reviews Immunology · doi:10.1038/nri981 · Cited by 661 · see citing works

Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity

Schiedner, G., Morral, N., Parks, R. J., Wu, Y., Koopmans, S. C., Langston, C., Graham, F. L., Beaudet, A. L., & Kochanek, S. (1998) · Nature Genetics · doi:10.1038/ng0298-180 · Cited by 648 · see citing works

Quantitative analysis of the packaging capacity of recombinant adeno-associated virus

Dong, J.-Y., Fan, P.-D., & Frizzell, R. A. (1996) · Human Gene Therapy · doi:10.1089/hum.1996.7.17-2101 · Cited by 583 · see citing works

Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses

Zhong, L., Li, B., Mah, C. S., Govindasamy, L., Agbandje-McKenna, M., Cooper, M., Herzog, R. W., Zolotukhin, I., Warrington, K. H., Weigel-Van Aken, K. A., et al. (2008) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0802866105 · Cited by 558 · see citing works

Increased apoptosis of Huntington disease lymphoblasts associated with repeat length-dependent mitochondrial depolarization

Sawa, A., Wiegand, G. W., Cooper, J. K., Margolis, R. L., Sharp, A. H., Lawler, J. F., Greenamyre, J. T., Snyder, S. H., & Ross, C. A. (1999) · Nature Medicine · doi:10.1038/13518 · Cited by 552 · see citing works

Stem Cells and Liver Regeneration

Duncan, A. W., Dorrell, C., & Grompe, M. (2009) · Gastroenterology · doi:10.1053/j.gastro.2009.05.044 · Cited by 547 · see citing works

The Hepatic Microcirculation: Mechanistic Contributions and Therapeutic Targets in Liver Injury and Repair

Vollmar, B., & Menger, M. D. (2009) · Physiological Reviews · doi:10.1152/physrev.00027.2008 · Cited by 505 · see citing works

Preliminary Communication

Bickel, H., GERRARD, J. W., & Hickmans, E. M. (1953) · The Lancet · doi:10.1016/s0140-6736(53)90473-5 · Cited by 499 · see citing works

Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas

Nault, J.-C., Datta, S., Imbeaud, S., Franconi, A., Mallet, M., Couchy, G., Letouzé, E., Pilati, C., Verret, B., Blanc, J.-F., et al. (2015) · Nature Genetics · doi:10.1038/ng.3389 · Cited by 489 · see citing works

Induction of Immune Tolerance to Coagulation Factor IX Antigen by In Vivo Hepatic Gene Transfer

Mingozzi, F., Liu, Y.-L., Dobrzynski, E., Kaufhold, A., Liu, J. H., Wang, Y., Arruda, V. R., High, K. A., & Herzog, R. W. (2003) · Journal of Clinical Investigation · doi:10.1172/jci16887 · Cited by 466 · see citing works

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

Lisowski, L., Dane, A. P., Chu, K., Zhang, Y., Cunningham, S. C., Wilson, E. M., Nygaard, S., Grompe, M., Alexander, I. E., & Kay, M. A. (2014) · Nature · doi:10.1038/nature12875 · Cited by 450 · see citing works

Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver

Nathwani, A. C., Gray, J. T., Ng, C. Y., Zhou, J., Spence, Y., Waddington, S. N., Tuddenham, E. G. D., Kemball‐Cook, G., McIntosh, J., Boon‐Spijker, M., et al. (2005) · Blood · doi:10.1182/blood-2005-10-4035 · Cited by 421 · see citing works

Packaging Capacity of Adeno-Associated Virus Serotypes: Impact of Larger Genomes on Infectivity and Postentry Steps

Grieger, J. C., & Samulski, R. J. (2005) · Journal of Virology · doi:10.1128/jvi.79.15.9933-9944.2005 · Cited by 419 · see citing works

Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors

Snyder, R. O., Miao, C., Meuse, L., Tubb, J., Donahue, B. A., Lin, H.-F., Stafford, D. W., Patel, S., Thompson, A. R., Nichols, T., et al. (1999) · Nature Medicine · doi:10.1038/4751 · Cited by 409 · see citing works

Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy

Chandler, R. J., LaFave, M. C., Varshney, G. K., Trivedi, N. S., Carrillo-Carrasco, N., Senac, J. S., Wu, W., Hoffmann, V., Elkahloun, A. G., Burgess, S. M., et al. (2015) · Journal of Clinical Investigation · doi:10.1172/jci79213 · Cited by 403 · see citing works

Exposing synonymous mutations

Hunt, R., Simhadri, V. L., Iandoli, M., Sauna, Z. E., & Kimchi‐Sarfaty, C. (2014) · Trends in Genetics · doi:10.1016/j.tig.2014.04.006 · Cited by 386 · see citing works

Orchestrating liver development

Gordillo, M., Evans, T., & Gouon-Evans, V. (2015) · Development · doi:10.1242/dev.114215 · Cited by 375 · cited 1× · see citing works

Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

Mingozzi, F., Anguela, X. M., Pavani, G., Chen, Y., Davidson, R. J., Hui, D. J., Yazicioglu, M., Elkouby, L., Hinderer, C. J., Faella, A., et al. (2013) · Science Translational Medicine · doi:10.1126/scitranslmed.3005795 · Cited by 355 · see citing works

Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

Mount, J. D., Herzog, R. W., Tillson, D. M., Goodman, S. A., Robinson, N., McCleland, M. L., Bellinger, D., Nichols, T. C., Arruda, V. R., Lothrop, C. D., et al. (2002) · Blood · doi:10.1182/blood.v99.8.2670 · Cited by 339 · see citing works

Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy

Jiang, H., Couto, L. B., Patarroyo-White, S., Liu, T., Nagy, D., Vargas, J. A., Zhou, S., Scallan, C. D., Sommer, J., Vijay, S., et al. (2006) · Blood · doi:10.1182/blood-2006-04-017913 · Cited by 330 · see citing works

Production of Recombinant Adeno-associated Virus Vectors Using Suspension HEK293 Cells and Continuous Harvest of Vector From the Culture Media for GMP FIX and FLT1 Clinical Vector

Grieger, J. C., Soltys, S. M., & Samulski, R. J. (2015) · Molecular Therapy · doi:10.1038/mt.2015.187 · Cited by 326 · see citing works

Adeno-associated viruses undergo substantial evolution in primates during natural infections

Gao, G., Alvira, M. R., Somanathan, S., Lü, Y., Vandenberghe, L. H., Rux, J. J., Calcedo, R., Sanmiguel, J. C., Abbas, Z., & Wilson, J. M. (2003) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0937739100 · Cited by 323 · see citing works

Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors

Donsante, A., Vogler, C. A., Muzyczka, N., Crawford, J., Barker, J. E., Flotte, T. R., Campbell‐Thompson, M., Daly, T., & Sands, M. S. (2001) · Gene Therapy · doi:10.1038/sj.gt.3301541 · Cited by 320 · see citing works

Structural and functional hepatocyte polarity and liver disease

Gissen, P., & Arias, I. M. (2015) · Journal of Hepatology · doi:10.1016/j.jhep.2015.06.015 · Cited by 316 · see citing works

Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice

Dominguez, E., Marais, T., Chatauret, N., Benkhelifa-Ziyyat, S., Duque, S., Ravassard, P., Carcenac, R., Astord, S., de Moura, A. P., Voit, T., et al. (2011) · Human Molecular Genetics · doi:10.1093/hmg/ddq514 · Cited by 298 · see citing works

Modulation of liver tolerance by conventional and nonconventional antigen-presenting cells and regulatory immune cells

Horst, A. K., Neumann, K., Diehl, L., & Tiegs, G. (2016) · Cellular &amp; Molecular Immunology · doi:10.1038/cmi.2015.112 · Cited by 295 · see citing works

Manufacturing of recombinant adeno-associated viral vectors for clinical trials

Clément, N., & Grieger, J. C. (2016) · Molecular Therapy - Methods &amp; Clinical Development · doi:10.1038/mtm.2016.2 · Cited by 286 · see citing works

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

McIntosh, J., Lenting, P. J., Rosales, C., Lee, D., Rabbanian, S., Raj, D., Patel, N., Tuddenham, E. G. D., Christophe, O. D., McVey, J. H., et al. (2013) · Blood · doi:10.1182/blood-2012-10-462200 · Cited by 278 · see citing works

Immune tolerance in liver disease

Crispe, I. N. (2014) · Hepatology · doi:10.1002/hep.27254 · Cited by 277 · see citing works

Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy

Niemeyer, G. P., Herzog, R. W., Mount, J., Arruda, V. R., Tillson, D. M., Hathcock, J., van Ginkel, F. W., High, K. A., & Lothrop, C. D. (2009) · Blood · doi:10.1182/blood-2008-10-181479 · Cited by 276 · see citing works

Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver

Mingozzi, F., Hasbrouck, N. C., Basner-Tschakarjan, E., Edmonson, S. A., Hui, D. J., Sabatino, D. E., Zhou, S., Wright, J. F., Jiang, H., Pierce, G. F., et al. (2007) · Blood · doi:10.1182/blood-2007-03-080093 · Cited by 254 · see citing works

Epitope Mapping of Human Anti-Adeno-Associated Virus Type 2 Neutralizing Antibodies: Implications for Gene Therapy and Virus Structure

Moskalenko, M., Chen, L., van Roey, M., Donahue, B. A., Snyder, R. O., McArthur, J. G., & Patel, S. D. (2000) · Journal of Virology · doi:10.1128/jvi.74.4.1761-1766.2000 · Cited by 253 · see citing works

Inclusion of the Hepatic Locus Control Region, an Intron, and Untranslated Region Increases and Stabilizes Hepatic Factor IX Gene Expression in Vivo but Not in Vitro

Miao, C. H., Ohashi, K., Patijn, G. A., Meuse, L. A., Ye, X., Thompson, A. R., & Kay, M. A. (2000) · Molecular Therapy · doi:10.1006/mthe.2000.0075 · Cited by 246 · see citing works

Humoral immune response to AAV

Calcedo, R., & Wilson, J. M. (2013) · Frontiers in Immunology · doi:10.3389/fimmu.2013.00341 · Cited by 243 · see citing works

"Lifespan" of Liver Cells

MacDonald, R. A. (1961) · Archives of Internal Medicine · doi:10.1001/archinte.1961.03620030023003 · Cited by 234 · see citing works

Assessing the potential for AAV vector genotoxicity in a murine model

Li, H., Malani, N., Hamilton, S. R., Schlachterman, A., Bussadori, G., Edmonson, S. A., Shah, R., Arruda, V. R., Mingozzi, F., Wright, J. F., et al. (2010) · Blood · doi:10.1182/blood-2010-08-302729 · Cited by 234 · see citing works

Characterization of Genome Integrity for Oversized Recombinant AAV Vector

Dong, B., Nakai, H., & Xiao, W. (2010) · Molecular Therapy · doi:10.1038/mt.2009.258 · Cited by 230 · see citing works

Sustained correction of bleeding disorder in hemophilia B mice by gene therapy

Wang, L., Takabe, K., Bidlingmaier, S. M., Ill, C. R., & Verma, I. M. (1999) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.96.7.3906 · Cited by 223 · see citing works

Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer

Cao, O., Dobrzynski, E., Wang, L., Nayak, S., Mingle, B., Terhorst, C., & Herzog, R. W. (2007) · Blood · doi:10.1182/blood-2007-02-073304 · Cited by 220 · see citing works

Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models

Davidoff, A. M., Gray, J. T., Ng, C. Y., Zhang, Y., Zhou, J., Spence, Y., Bakar, Y., & Nathwani, A. C. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2004.12.022 · Cited by 218 · see citing works

Immune responses to adeno-associated virus and its recombinant vectors.

Sun, J. Y., Anand-Jawa, V, Chatterjee, S., & Wong, K. K. (2003) · Gene Therapy · doi:10.1038/sj.gt.3302039 · Cited by 215 · see citing works

Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease

Messinger, Y. H., Mendelsohn, N. J., Rhead, W. J., Dimmock, D., Hershkovitz, E., Champion, M., Jones, S. A., Olson, R., White, A. L., Wells, C., et al. (2012) · Genetics in Medicine · doi:10.1038/gim.2011.4 · Cited by 214 · see citing works

Adeno-Associated Virus Type 2 VP2 Capsid Protein Is Nonessential and Can Tolerate Large Peptide Insertions at Its N Terminus

Warrington, K. H., Gorbatyuk, O. S., Harrison, J. K., Opie, S. R., Zolotukhin, S., & Muzyczka, N. (2004) · Journal of Virology · doi:10.1128/jvi.78.12.6595-6609.2004 · Cited by 205 · see citing works

Manufacturing and characterizing AAV-based vectors for use in clinical studies

Wright, J. F. (2008) · Gene Therapy · doi:10.1038/gt.2008.65 · Cited by 205 · see citing works

Loss of Siglec expression on T lymphocytes during human evolution

Nguyen, D. H., Hurtado-Ziola, N., Gagneux, P., & Varki, A. (2006) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0510484103 · Cited by 204 · see citing works

Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs

Jiang, H., Lillicrap, D., Patarroyo-White, S. L., Liu, T., Qian, X., Scallan, C. D., Powell, S., Keller, T., McMurray, M., Labelle, A., et al. (2006) · Blood · doi:10.1182/blood-2005-12-5115 · Cited by 200 · see citing works

A largely random AAV integration profile after LPLD gene therapy

Kaeppel, C., Beattie, S. G., Fronza, R., van Logtenstein, R., Salmon, F., Schmidt, S., Wolf, S., Nowrouzi, A., Glimm, H., Kalle, C. V., et al. (2013) · Nature Medicine · doi:10.1038/nm.3230 · Cited by 190 · see citing works

Recombinant AAV Integration Is Not Associated with Hepatic Genotoxicity in Nonhuman Primates and Patients

Gil-Farina, I., Fronza, R., Kaeppel, C., Lopez-Franco, E., Ferreira, V., D'Avola, D., Benito, A., Prieto, J., Petry, H., Gonzalez-Aseguinolaza, G., et al. (2016) · Molecular Therapy · doi:10.1038/mt.2016.52 · Cited by 179 · see citing works

Adeno-Associated Virus Integration: Virus versus Vector

Smith, R. H. (2008) · Gene Therapy · doi:10.1038/gt.2008.55 · Cited by 178 · see citing works

Displaying High-affinity Ligands on Adeno-associated Viral Vectors Enables Tumor Cell-specific and Safe Gene Transfer

Münch, R. C., Janicki, H., Völker, I., Rasbach, A., Hallek, M. J., Büning, H., & Buchholz, C. J. (2012) · Molecular Therapy · doi:10.1038/mt.2012.186 · Cited by 176 · see citing works

Sustained Expression of Therapeutic Level of Factor IX in Hemophilia B Dogs by AAV-Mediated Gene Therapy in Liver

Wang, L., Nichols, T. C., Read, M. S., Bellinger, D. A., & Verma, I. M. (2000) · Molecular Therapy · doi:10.1006/mthe.2000.0031 · Cited by 176 · see citing works

Muscle-Directed Gene Transfer and Transient Immune Suppression Result in Sustained Partial Correction of Canine Hemophilia B Caused by a Null Mutation

Herzog, R. W., Mount, J. D., Arruda, V. R., High, K. A., & Lothrop, C. D. (2001) · Molecular Therapy · doi:10.1006/mthe.2001.0442 · Cited by 176 · see citing works

Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors.

Cunningham, S., Dane, A. P., Spinoulas, A., & Alexander, I. (2008) · Molecular Therapy · doi:10.1038/mt.2008.72 · Cited by 175 · see citing works

Sustained correction of disease in naive and AAV2-pretreated hemophilia b dogs: AAV2/8-mediated, liver-directed gene therapy

Wang, L., Calcedo, R., Nichols, T. C., Bellinger, D. A., Dillow, A., Verma, I. M., & Wilson, J. M. (2005) · Blood · doi:10.1182/blood-2004-10-3867 · Cited by 173 · see citing works

Biology of AAV Serotype Vectors in Liver-Directed Gene Transfer to Nonhuman Primates

Gao, G., Lü, Y., Calcedo, R., Grant, R. L., Bell, P. L., Wang, L., Figueredo, J. M., Lock, M. W., & Wilson, J. M. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.08.017 · Cited by 170 · see citing works

Use of a Liver-Specific Promoter Reduces Immune Response to the Transgene in Adenoviral Vectors

Pastore, L., Morral, N., Zhou, H., Garcia, R. G. F., Parks, R. J., Kochanek, S., Graham, F. L., Lee, B., & Beaudet, A. L. (1999) · Human Gene Therapy · doi:10.1089/10430349950017455 · Cited by 168 · see citing works

Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery

Chicoine, L., Montgomery, C., Bremer, W., Shontz, K., Griffin, D., Heller, K., Lewis, S., Malik, V, Grose, W., Shilling, C., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2013.244 · Cited by 167 · see citing works

Efficient Serotype-Dependent Release of Functional Vector into the Culture Medium During Adeno-Associated Virus Manufacturing

Vandenberghe, L. H., Xiao, R., Lock, M., Lin, J., Korn, M., & Wilson, J. M. (2010) · Human Gene Therapy · doi:10.1089/hum.2010.107 · Cited by 151 · see citing works

Analysis of Tumors Arising in Male B6C3F1 Mice with and without AAV Vector Delivery to Liver

Bell, P. L., Moscioni, A. D., McCarter, R., Wu, D., Gao, G., Hoang, A., Sanmiguel, J. C., Sun, X., Wivel, N. A., Raper, S. E., et al. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.03.008 · Cited by 150 · see citing works

DLK1-DIO3 Genomic Imprinted MicroRNA Cluster at 14q32.2 Defines a Stemlike Subtype of Hepatocellular Carcinoma Associated with Poor Survival

Luk, J. M., Burchard, J., Zhang, C., Liu, A. M., Wong, K. F., Shek, F. H., Lee, N. P. Y., Fan, S. T., Poon, R. T. P., Ivanovska, I. L., et al. (2011) · Journal of Biological Chemistry · doi:10.1074/jbc.m111.229831 · Cited by 149 · see citing works

Monogenic diseases that can be cured by liver transplantation

FAGIUOLI, S., Daina, E., D’Antiga, L., Colledan, M., & Remuzzi, G. (2013) · Journal of Hepatology · doi:10.1016/j.jhep.2013.04.004 · Cited by 144 · see citing works

Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids

Chamberlain, K., Riyad, J. M., & Weber, T. (2016) · Human Gene Therapy Methods · doi:10.1089/hgtb.2015.140 · Cited by 141 · cited 1× · see citing works

Safe, Efficient, and Reproducible Gene Therapy of the Brain in the Dog Models of Sanfilippo and Hurler Syndromes

Ellinwood, N. M., Ausseil, J., Desmaris, N., Bigou, S., Liu, S., Jens, J. K., Snella, E. M., Mohammed, E. E., Thomson, C., Raoul, S., et al. (2010) · Molecular Therapy · doi:10.1038/mt.2010.265 · Cited by 137 · see citing works

Systematic Evaluation of AAV Vectors for Liver directed Gene Transfer in Murine Models

Wang, L., Wang, H., Bell, P. L., McCarter, R., He, J., Calcedo, R., Vandenberghe, L. H., Morizono, H., Batshaw, M. L., & Wilson, J. M. (2009) · Molecular Therapy · doi:10.1038/mt.2009.246 · Cited by 135 · see citing works

Design and Packaging of Adeno-Associated Virus Gene Targeting Vectors

Hirata, R. K., & Russell, D. W. (2000) · Journal of Virology · doi:10.1128/jvi.74.10.4612-4620.2000 · Cited by 133 · see citing works

Risk and Prevention of Anti-factor IX Formation in AAV-Mediated Gene Transfer in the Context of a Large Deletion of F9

Fields, P. A., Arruda, V. R., Armstrong, E., Chu, K., Mingozzi, F., Hagstrom, J. N., Herzog, R. W., & High, K. A. (2001) · Molecular Therapy · doi:10.1006/mthe.2001.0441 · Cited by 127 · see citing works

Large-scale recombinant adeno-associated virus production

Kotin, R. M. (2011) · Human Molecular Genetics · doi:10.1093/hmg/ddr141 · Cited by 123 · see citing works

Novel Forms of B‐Domain‐Deleted Recombinant Factor VIII Molecules: Construction and Biochemical Characterization

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Codon and mRNA sequence optimization of microdystrophin transgenes improves expression and physiological outcome in dystrophic mdx mice following AAV2/8 gene transfer

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Recent advancements in exon-skipping therapies using antisense oligonucleotides and genome editing for the treatment of various muscular dystrophies

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Single amino acid insertion allows functional transduction of murine hepatocytes with human liver tropic AAV capsids

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Liver-specific drug delivery platforms: Applications for the treatment of alcohol-associated liver disease

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Low-Dose Gene Therapy for Murine PKU Using Episomal Naked DNA Vectors Expressing PAH from Its Endogenous Liver Promoter

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Retrograde axonal transport property of adeno-associated virus and its possible application in future

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Therapeutic editing of hepatocyte genome in vivo

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Insulin‐like growth factor binding protein 7 accelerates hepatic steatosis and insulin resistance in non‐alcoholic fatty liver disease

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miRNA Pathway Alteration in Response to Non-Coding RNA Delivery in Viral Vector-Based Gene Therapy

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Advances in Delivery Vectors for Gene Therapy in Liver Cancer

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Gene Therapy with an Adeno-Associated Viral Vector Expressing Human Interleukin-2 Alters Immune System Homeostasis in Humanized Mice

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Characterization of IL-21-expressing recombinant hepatitis B virus (HBV) as a therapeutic agent targeting persisting HBV infection

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The intersection of vector biology, gene therapy, and hemophilia

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Multivalent Targeting of the Asialoglycoprotein Receptor by Virus‐Like Particles

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Dexamethasone Transiently Enhances Transgene Expression in the Liver When Administered at Late-Phase Post Long-Term Adeno-Associated Virus Transduction

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Viral Vectors in Gene Replacement Therapy

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Liver-specific insulin receptor isoform A expression enhances hepatic glucose uptake and ameliorates liver steatosis in a mouse model of diet-induced obesity

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Guanylin and uroguanylin mRNA expression is increased following Roux-en-Y gastric bypass, but guanylins do not play a significant role in body weight regulation and glycemic control

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Biodistribution of adeno‐associated virus type 2 carrying multi‐characteristic opsin in dogs following intravitreal injection

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Long‐term functional correction of cystathionine β‐synthase deficiency in mice by adeno‐associated viral gene therapy

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Direct recognition of hepatocyte-expressed MHC class I alloantigens is required for tolerance induction

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Visualization of the therapeutic efficacy of a gene correction approach in Wilson’s disease by laser-ablation inductively coupled mass spectrometry

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Application of in-vitro-cultured primary hepatocytes to evaluate species translatability and AAV transduction mechanisms of action

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Insights into Gene Therapy for Urea Cycle Defects by Mathematical Modeling

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Analysis of hepatic and retinal cell microRNAome during AAV infection reveals their diverse impact on viral transduction and cellular physiology

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Jugular Vein Injection of High-Titer Lentiviral Vectors Does Not Transduce the Aorta—Brief Report

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Adeno-associated virus-vectored delivery of HIV biologics: the promise of a “single-shot” functional cure for HIV infection

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BCKDHA-BCKDHB digenic gene therapy restores metabolic homeostasis in two mouse models and a calf with classic maple syrup urine disease

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Practical Implications of Factor IX Gene Transfer for Individuals with Hemophilia B: A Clinical Perspective

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ZNF469 is a profibrotic regulator of extracellular matrix in hepatic stellate cells

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Improving AAV Production Yield and Quality for Different Serotypes Using Distinct Processing Methods

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Comprehensive analysis of adverse events associated with onasemnogene abeparvovec (Zolgensma) in spinal muscular atrophy patients: insights from FAERS database

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Characterizing the Content and Structure of AAV Capsids by Size Exclusion Chromatography and Orbitrap-Based Charge Detection-Mass Spectrometry

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Local and systemic administration of AAV vectors with alphaherpesvirus latency-associated promoter 2 drives potent transgene expression in mouse liver, kidney, and skeletal muscle

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Preclinical efficacy and safety of adeno-associated virus 5 alpha-galactosidase: A gene therapy for Fabry disease

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Gene Therapy Strategies for Hepatocellular Carcinoma (HCC): Current Landscape and Future Directions

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iPSC-hepatocyte organoids as a novel platform to predict AAV gene therapy efficacy

Berreur, E., Lazzaroni, G., Roth, C., Zihlmann, M., Stirn, M., Matheis, R., Xicluna, R., Breous-Nystrom, E., & Roth, A. B. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101467 · Cited by 9 · see citing works

Gene Therapy for Hemophilia A: Where We Stand

Zhou, M., Hu, Z., Zhang, C., Wu, L., Li, Z., & Liang, D. (2020) · Current Gene Therapy · doi:10.2174/1566523220666200806110849 · Cited by 8 · see citing works

Novel Coagulation Factor VIII Gene Therapy in a Mouse Model of Hemophilia A by Lipid-Coated Fe3O4 Nanoparticles

Kao, Y., Chen, Y., Fan, H., Tsai, T., Cheng, S., Lai, P., Chen, J., & Chen, C. (2021) · Biomedicines · doi:10.3390/biomedicines9091116 · Cited by 8 · see citing works

Promoter CAG is more efficient than hepatocyte‑targeting TBG for transgene expression via rAAV8 in liver tissues

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Mutagenesis and Carcinogenesis Risk Evaluation for AAV and Lentiviral Gene Therapies

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Safety and feasibility of the gene transfer of hemopexin for conditions with increased free heme

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Inherently Reduced Expression of ASC Restricts Caspase-1 Processing in Hepatocytes and Promotes Plasmodium Infection

Marques-da-Silva, C., Schmidt-Silva, C., Baptista, R. P., & Kurup, S. P. (2023) · The Journal of Immunology · doi:10.4049/jimmunol.2300440 · Cited by 7 · see citing works

In-Depth Characterization of a Mifepristone-Regulated Expression System for AAV5-Mediated Gene Therapy in the Liver

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Peripheral ProBDNF Delivered by an AAV Vector to the Muscle Triggers Depression-Like Behaviours in Mice

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Silencing hepatic PCSK9 via novel chimeric AAV8 mitigates the progression of atherosclerosis by inhibiting inflammation in ApoE−/− mice

Chen, X., Sun, M., Ma, X., Ma, Y., & Chen, B. (2024) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2024.101390 · Cited by 6 · see citing works

Administration and detection of a multi-target rAAV gene doping vector in horses using multiple matrices and molecular techniques

Maniego, J., Harding, C., Habershon‐Butcher, J., Hincks, P. R., & Ryder, E. (2024) · Gene Therapy · doi:10.1038/s41434-024-00462-0 · Cited by 6 · see citing works

Age‐stratified adeno‐associated virus serotype 3 neutralizing and total antibody prevalence in hemophilia A patients from India

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The linkage between microRNA and cancer and its delivery as cancer therapy: A mini-review

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Hemophilia Healing with AAV: Navigating the Frontier of Gene Therapy

Khan, S. U., Khan, M. U., Suleman, M., Inam, A., & Din, M. A. U. (2024) · Current Gene Therapy · doi:10.2174/0115665232279893231228065540 · Cited by 6 · see citing works

A Versatile Adeno-Associated Viral Vector Cross-Linking Platform Capable of Tuning Cellular Tropisms and Simultaneously Inducing Solid-Phase Gene Delivery

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The deLIVERed promises of gene therapy: Past, present, and future of liver-directed gene therapy

Puzzo, F., & Kay, M. A. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.03.041 · Cited by 24 · see citing works

Current and Emerging Issues in Adeno-Associated Virus Vector-Mediated Liver-Directed Gene Therapy

Piccolo, P., & Brunetti‐Pierri, N. (2024) · Human Gene Therapy · doi:10.1089/hum.2024.179 · Cited by 14 · see citing works

Liver directed adeno‐associated viral vectors to treat metabolic disease

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Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects

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AAV-mediated gene therapy for liver diseases: the prime candidate for clinical application?

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