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Drawing a high-resolution functional map of adeno-associated virus capsid by massively parallel sequencing

Adachi, K., Enoki, T., Kawano, Y., Veraz, M., & Nakai, H. (2014) · Nature Communications · doi:10.1038/ncomms4075 · Cited by 149 · see citing works

Versatility of AAV vectors for retinal gene transfer

Surace, E. M., & Auricchio, A. (2007) · Vision Research · doi:10.1016/j.visres.2007.07.027 · Cited by 145 · see citing works

The advent of AAV9 expands applications for brain and spinal cord gene delivery

Dayton, R. D., Wang, D. B., & Klein, R. L. (2012) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.2012.681463 · Cited by 144 · see citing works

Novel random peptide libraries displayed on AAV serotype 9 for selection of endothelial cell-directed gene transfer vectors

Varadi, K., Michelfelder, S., Korff, T., Hecker, M., Trepel, M., Katus, H. A., Kleinschmidt, J. A., & Müller, O. J. (2011) · Gene Therapy · doi:10.1038/gt.2011.143 · Cited by 145 · see citing works

Gene Therapy for Heart Failure

Tilemann, L. M., Ishikawa, K., Weber, T., & Hajjar, R. J. (2012) · Circulation Research · doi:10.1161/circresaha.111.252981 · Cited by 140 · see citing works

Adeno-Associated Virus Vectors as Therapeutic and Investigational Tools in the Cardiovascular System

Zacchigna, S., Zentilin, L., & Giacca, M. (2014) · Circulation Research · doi:10.1161/circresaha.114.302331 · Cited by 140 · see citing works

Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8

Natkunarajah, M., Trittibach, P., McIntosh, J., Duran, Y., Barker, S. E., Smith, A. J., Nathwani, A. C., & Ali, R. R. (2008) · Gene Therapy · doi:10.1038/sj.gt.3303074 · Cited by 138 · see citing works

Identification of the Galactose Binding Domain of the Adeno-Associated Virus Serotype 9 Capsid

Bell, C. L., Gurda, B. L., Vliet, K. V., Agbandje‐McKenna, M., & Wilson, J. M. (2012) · Journal of Virology · doi:10.1128/jvi.00448-12 · Cited by 138 · see citing works

Comparative Cardiac Gene Delivery of Adeno‐Associated Virus Serotypes 1–9 reveals that AAV6 Mediates the Most Efficient Transduction in Mouse Heart

Zincarelli, C., Soltys, S., Rengo, G., Koch, W. J., & Rabinowitz, J. E. (2010) · Clinical and Translational Science · doi:10.1111/j.1752-8062.2010.00190.x · Cited by 137 · see citing works

Adeno-Associated Viral Vectors and Their Redirection to Cell-Type Specific Receptors

Michelfelder, S., & Trepel, M. (2009) · Advances in genetics · doi:10.1016/s0065-2660(09)67002-4 · Cited by 134 · see citing works

AAV-Mediated Liver-Directed Gene Therapy

Sands, M. S. (2011) · Methods in molecular biology · doi:10.1007/978-1-61779-370-7_6 · Cited by 134 · see citing works

Circulating miR-103a-3p contributes to angiotensin II-induced renal inflammation and fibrosis via a SNRK/NF-κB/p65 regulatory axis

Lu, Q., Ma, Z., Ye, D., Bédarida, T., Chen, L., Xie, Z., Song, P., & Zou, M. (2019) · Nature Communications · doi:10.1038/s41467-019-10116-0 · Cited by 136 · see citing works

microRNA122-regulated transgene expression increases specificity of cardiac gene transfer upon intravenous delivery of AAV9 vectors

Geisler, A., Jungmann, A., Kurreck, J., Poller, W. C., Katus, H. A., Vetter, R., Fechner, H., & Müller, O. J. (2010) · Gene Therapy · doi:10.1038/gt.2010.141 · Cited by 132 · see citing works

Intravenous Gene Therapy With PIM-1 Via a Cardiotropic Viral Vector Halts the Progression of Diabetic Cardiomyopathy Through Promotion of Prosurvival Signaling

Katare, R., Caporali, A., Zentilin, L., Avolio, E., Sala‐Newby, G. B., Oikawa, A., Cesselli, D., Beltrami, A. P., Giacca, M., Emanueli, C., et al. (2011) · Circulation Research · doi:10.1161/circresaha.110.239111 · Cited by 130 · see citing works

Genome editing with CRISPR/Cas9 in postnatal mice corrects PRKAG2 cardiac syndrome

Xie, C., Zhang, Y., Song, L., Luo, J., Qi, W., Hu, J., Lu, D., Yang, Z. H., Zhang, J., Xiao, J., et al. (2016) · Cell Research · doi:10.1038/cr.2016.101 · Cited by 125 · see citing works

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

Foster, H., Sharp, P. S., Athanasopoulos, T., Trollet, C., Graham, I. R., Foster, K., Wells, D. J., & Dickson, G. (2008) · Molecular Therapy · doi:10.1038/mt.2008.186 · Cited by 119 · see citing works

CRISPR Modeling and Correction of Cardiovascular Disease

Liu, N., & Olson, E. N. (2022) · Circulation Research · doi:10.1161/circresaha.122.320496 · Cited by 118 · see citing works

Telomerase gene therapy rescues telomere length, bone marrow aplasia, and survival in mice with aplastic anemia

Bär, C., Povedano, J. M., Serrano, R., Benitez-Buelga, C., Popkes, M., Formentini, I., Bobadilla, M., Bosch, F., & Blasco, M. A. (2016) · Blood · doi:10.1182/blood-2015-08-667485 · Cited by 116 · see citing works

Adeno-Associated Virus Serotype-Specific Inverted Terminal Repeat Sequence Role in Vector Transgene Expression

Earley, L. F., Conatser, L. M., Lue, V. M., Dobbins, A. L., Li, C., Hirsch, M. L., & Samulski, R. J. (2020) · Human Gene Therapy · doi:10.1089/hum.2019.274 · Cited by 116 · see citing works

MiR-30c/PGC-1β protects against diabetic cardiomyopathy via PPARα

Yin, Z., Zhao, Y., He, M., Li, H., Fan, J., Nie, X., Yan, M., Chen, C., & Wang, D. W. (2019) · Cardiovascular Diabetology · doi:10.1186/s12933-019-0811-7 · Cited by 116 · see citing works

Adeno-associated viral vectors do not efficiently target muscle satellite cells

Arnett, A. L. H., Konieczny, P., Ramos, J. N., Hall, J. K., Odom, G., Yablonka‐Reuveni, Z., Chamberlain, J. R., & Chamberlain, J. S. (2014) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2014.38 · Cited by 110 · see citing works

Efficient precise in vivo base editing in adult dystrophic mice

Xu, L. M., Zhang, C., Bailin, P. L., Wang, P., Gao, Y., Mohler, P. J., Mokadam, N. A., Ma, J., Arnold, W. D., & Han, R. (2021) · Nature Communications · doi:10.1038/s41467-021-23996-y · Cited by 109 · see citing works

Tissue specific promoters improve specificity of AAV9 mediated transgene expression following intra-vascular gene delivery in neonatal mice

Pacak, C. A., Sakai, Y., Thattaliyath, B. D., Mah, C. S., & Byrne, B. J. (2008) · Genetic Vaccines and Therapy · doi:10.1186/1479-0556-6-13 · Cited by 111 · see citing works

Injection of a recombinant AAV serotype 2 into canine skeletal muscles evokes strong immune responses against transgene products

Yuasa, K., Yoshimura, M., Urasawa, N., Ohshima, S., Howell, J. M., Nakamura, A., Hijikata, T., Miyagoe‐Suzuki, Y., & Takeda, S. (2007) · Gene Therapy · doi:10.1038/sj.gt.3302984 · Cited by 107 · see citing works

VEGF overexpression via adeno‐associated virus gene transfer promotes skeletal muscle regeneration and enhances muscle function in mdx mice

Messina, S., Mazzeo, A. T., Bitto, A., Aguennouz, M., Migliorato, A., Pasquale, M. G. D., Minutoli, L., Altavilla, D., Zentilin, L., Giacca, M., et al. (2007) · The FASEB Journal · doi:10.1096/fj.07-8459com · Cited by 107 · see citing works

Comparison of Gene Delivery to the Kidney by Adenovirus, Adeno-Associated Virus, and Lentiviral Vectors After Intravenous and Direct Kidney Injections

Rubin, J. D., Nguyen, T. V., Allen, K. L., Ayasoufi, K., & Barry, M. A. (2019) · Human Gene Therapy · doi:10.1089/hum.2019.127 · Cited by 105 · see citing works

Engraftment of a Galactose Receptor Footprint onto Adeno-associated Viral Capsids Improves Transduction Efficiency

Shen, S., Horowitz, E. D., Troupes, A. N., Brown, S. M., Pulicherla, N., Samulski, R. J., Agbandje‐McKenna, M., & Asokan, A. (2013) · Journal of Biological Chemistry · doi:10.1074/jbc.m113.482380 · Cited by 103 · see citing works

GJA1-20k Arranges Actin to Guide Cx43 Delivery to Cardiac Intercalated Discs

Basheer, W. A., Xiao, S., Epifantseva, I., Fu, Y., Kléber, A. G., Hong, T., & Shaw, R. M. (2017) · Circulation Research · doi:10.1161/circresaha.117.311955 · Cited by 104 · see citing works

Correction of Pathological Accumulation of Glycosaminoglycans in Central Nervous System and Peripheral Tissues of MPSIIIA Mice Through Systemic AAV9 Gene Transfer

Ruzo, A., Marcó, S., García, M., Villacampa, P., Ribera, A., Ayuso, E., Maggioni, L., Mingozzi, F., Haurigot, V., & Bosch, F. (2012) · Human Gene Therapy · doi:10.1089/hum.2012.029 · Cited by 104 · see citing works

Systemic delivery of adeno-associated viral vectors

Duan, D. (2016) · Current Opinion in Virology · doi:10.1016/j.coviro.2016.07.006 · Cited by 103 · see citing works

Rescue of cardiomyopathy through U7snRNA‐mediated exon skipping in Mybpc3‐targeted knock‐in mice

Gedicke, C., Behrens‐Gawlik, V., Reischmann, S., Geertz, B., Stimpel, D., Weinberger, F., Schlossarek, S., Précigout, G., Braren, I., Thomas Eschenhagen, A., et al. (2013) · EMBO Molecular Medicine · doi:10.1002/emmm.201202168 · Cited by 101 · see citing works

Enhancing Muscle Membrane Repair by Gene Delivery of MG53 Ameliorates Muscular Dystrophy and Heart Failure in δ-Sarcoglycan-deficient Hamsters

He, B., Tang, R., Weisleder, N. L., Xiao, B., Yuan, Z., Cai, C., Hua Zhu, C., Lin, P., Qiao, C., Li, J., et al. (2012) · Molecular Therapy · doi:10.1038/mt.2012.5 · Cited by 98 · see citing works

Comparative analysis of in vivo and in vitro AAV vector transduction in the neonatal mouse retina: Effects of serotype and site of administration

Pang, J.-J., Lauramore, A., Deng, W.-T., Li, Q., Doyle, T. J., Chiodo, V., Li, J., & Hauswirth, W. W. (2008) · Vision Research · doi:10.1016/j.visres.2007.08.009 · Cited by 97 · see citing works

AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice

Hakim, C. H., Wasala, N. B., Nelson, C. E., Wasala, L. P., Yue, Y., Louderman, J. A., Lessa, T. B., Dai, A., Zhang, K., Jenkins, G. J., et al. (2018) · JCI Insight · doi:10.1172/jci.insight.124297 · Cited by 99 · see citing works

Adeno-Associated Virus Serotype-9 Microdystrophin Gene Therapy Ameliorates Electrocardiographic Abnormalities in mdx Mice

Bostick, B., Yue, Y., Lai, Y., Long, C., Li, D., & Duan, D. (2008) · Human Gene Therapy · doi:10.1089/hum.2008.058 · Cited by 98 · see citing works

The potential of adeno-associated viral vectors for gene delivery to muscle tissue

Wang, D., Li, Z., Nahid, M. A., & Gao, G. (2014) · Expert Opinion on Drug Delivery · doi:10.1517/17425247.2014.871258 · Cited by 97 · see citing works

Cardio-specific long-term gene expression in a porcine model after selective pressure-regulated retroinfusion of adeno-associated viral (AAV) vectors

Raake, P. W. J., Hinkel, R., Müller, S., Delker, S., Kreuzpointner, R., Kupatt, C., Katus, H. A., Kleinschmidt, J. A., Boekstegers, P., & Müller, O. J. (2007) · Gene Therapy · doi:10.1038/sj.gt.3303035 · Cited by 96 · see citing works

Gene Therapy Mediated Partial Reprogramming Extends Lifespan and Reverses Age-Related Changes in Aged Mice.

Macip, C. C., Hasan, R., Hoznek, V., Kim, J., Metzger, L. E., Sethna, S., & Davidsohn, N. (2023) · doi:10.1101/2023.01.04.522507 · Cited by 95 · see citing works

MicroRNA-regulated viral vectors for gene therapy

Geisler, A., & Fechner, H. (2016) · World Journal of Experimental Medicine · doi:10.5493/wjem.v6.i2.37 · Cited by 96 · see citing works

Rapid and highly efficient inducible cardiac gene knockout in adult mice using AAV-mediated expression of Cre recombinase

Werfel, S., Jungmann, A., Lehmann, L., Ksienzyk, J., Bekeredjian, R., Kaya, Z., Leuchs, B., Nordheim, A., Backs, J., Engelhardt, S. H., et al. (2014) · Cardiovascular Research · doi:10.1093/cvr/cvu174 · Cited by 94 · see citing works

Introducing Genes into Mammalian Cells: Viral VectorsPreprint

Sena‐Esteves, M., & Gao, G. (2020) · Cold Spring Harbor Protocols · doi:10.1101/pdb.top095513 · Cited by 89 · see citing works

Transduction of Nonhuman Primate Brain with Adeno-Associated Virus Serotype 1: Vector Trafficking and Immune Response

Hadaczek, P., Forsayeth, J., Mirek, H., Munson, K., Bringas, J., Pivirotto, P., L McBride, J., Davidson, B. L., & Bankiewicz, K. S. (2009) · Human Gene Therapy · doi:10.1089/hum.2008.151 · Cited by 90 · see citing works

Structure of AAV-DJ, a Retargeted Gene Therapy Vector: Cryo-Electron Microscopy at 4.5 Å Resolution

Lerch, T. F., O’Donnell, J., Meyer, N., Xie, Q., Taylor, K. A., Stagg, S. M., & Chapman, M. S. (2012) · Structure · doi:10.1016/j.str.2012.05.004 · Cited by 89 · see citing works

doi:10.1016/j.ymthe.2006.03.014 ARTICLE Robust Systemic Transduction with AAV9 Vectors in Mice: Efficient Global Cardiac Gene Transfer Superior to That of AAV8

Inagaki, K., Fuess, S., Storm, T. A., Gibson, G. A., McTiernan, C. F., Kay, M. A., & Nakai, H. (2006)

AAV-8 Is More Efficient than AAV-9 in Transducing Neonatal Dog Heart

Pan, X., Yue, Y., Zhang, K., Hakim, C. H., Kodippili, K., McDonald, T., & Duan, D. (2015) · Human Gene Therapy Methods · doi:10.1089/hgtb.2014.128 · Cited by 26 · see citing works

Recombinant Adeno-Associated Virus Serotype 9 Leads to Preferential Cardiac Transduction In Vivo

Pacak, C. A., Mah, C. S., Thattaliyath, B. D., Conlon, T. J., Lewis, M. A., Cloutier, D. E., Zolotukhin, I., Tarantal, A. F., & Byrne, B. J. (2006) · Circulation Research · doi:10.1161/01.res.0000237661.18885.f6 · Cited by 406 · see citing works

AAV Vectors for Efficient Gene Delivery to Rodent Hearts

López-Gordo, E., Kohlbrenner, E., Katz, M. G., & Weber, T. (2019) · Methods in molecular biology · doi:10.1007/978-1-4939-9139-6_19 · Cited by 10 · see citing works

Selective tropism of the recombinant adeno‐associated virus 9 serotype for rat cardiac tissue

Qi, Y., Liu, X., Li, H., Shenoy, V., Li, Q., Hauswirth, W. W., Sumners, C., & Katovich, M. J. (2009) · The Journal of Gene Medicine · doi:10.1002/jgm.1404 · Cited by 15 · see citing works