rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice

X chromosome-linked muscular dystrophy (mdx) in the mouse

Bulfield, G., Siller, W. G., Wight, P. A., & Moore, K. J. (1984) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.81.4.1189 · Cited by 1,765

Systemic delivery of genes to striated muscles using adeno-associated viral vectors

Gregorevic, P., Blankinship, M. J., Allen, J. M., Crawford, R. W., Meuse, L., Miller, D. G., Russell, D. W., & Chamberlain, J. S. (2004) · Nature Medicine · doi:10.1038/nm1085 · Cited by 620

Dystrophin Immunity in Duchenne's Muscular Dystrophy

Mendell, J. R., Campbell, K., Rodino-Klapac, L., Sahenk, Z., Shilling, C., Lewis, S., Bowles, D., Gray, S., Li, C., Galloway, G., et al. (2010) · New England Journal of Medicine · doi:10.1056/nejmoa1000228 · Cited by 655

Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy

Duan, D. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.07.011 · Cited by 489

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

Bowles, D. E., McPhee, S. W., Li, C., Gray, S. J., Samulski, J. J., Camp, A. S., Li, J., Wang, B., Monahan, P. E., Rabinowitz, J. E., et al. (2012) · Molecular Therapy · doi:10.1038/mt.2011.237 · Cited by 400

Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

Bengtsson, N. E., Hall, J. K., Odom, G. L., Phelps, M. P., Andrus, C. R., Hawkins, R. D., Hauschka, S. D., Chamberlain, J. R., & Chamberlain, J. S. (2017) · Nature Communications · doi:10.1038/ncomms14454 · Cited by 387

The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice

Zhu, J., Huang, X., & Yang, Y. (2009) · Journal of Clinical Investigation · doi:10.1172/jci37607 · Cited by 330

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

Le Guiner, C., Servais, L., Montus, M., Larcher, T., Fraysse, B., Moullec, S., Allais, M., François, V., Dutilleul, M., Malerba, A., et al. (2017) · Nature Communications · doi:10.1038/ncomms16105 · Cited by 246

Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle

Salva, M. Z., Himeda, C. L., Tai, P. W., Nishiuchi, E., Gregorevic, P., Allen, J. M., Finn, E. E., Nguyen, Q. G., Blankinship, M. J., Meuse, L., et al. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300027 · Cited by 232

Follistatin-mediated skeletal muscle hypertrophy is regulated by Smad3 and mTOR independently of myostatin

Winbanks, C. E., Weeks, K. L., Thomson, R. E., Sepulveda, P. V., Beyer, C., Qian, H., Chen, J. L., Allen, J. M., Lancaster, G. I., Febbraio, M. A., et al. (2012) · Journal of Cell Biology · doi:10.1083/jcb.201109091 · Cited by 218

A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8

Monteilhet, V., Saheb, S., Boutin, S., Leborgne, C., Veron, P., Montus, M.-F., Moullier, P., Benveniste, O., & Masurier, C. (2011) · Molecular Therapy · doi:10.1038/mt.2011.108 · Cited by 206

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape

Mingozzi, F., & High, K. A. (2017) · Annual Review of Virology · doi:10.1146/annurev-virology-101416-041936 · Cited by 206

Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease

Rodino‐Klapac, L. R., Haidet, A. M., Kota, J., Handy, C., Kaspar, B. K., & Mendell, J. R. (2009) · Muscle & Nerve · doi:10.1002/mus.21244 · Cited by 194

A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs

Yue, Y., Ghosh, A., Long, C., Bostick, B., Smith, B. F., Kornegay, J. N., & Duan, D. (2008) · Molecular Therapy · doi:10.1038/mt.2008.207 · Cited by 173

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

Production of adeno-associated virus vectors for in vitro and in vivo applications

Kimura, T., Ferran, B., Tsukahara, Y., Shang, Q., Desai, S., Fedoce, A., Pimentel, D. R., Luptak, I., Adachi, T., Ido, Y., et al. (2019) · Scientific Reports · doi:10.1038/s41598-019-49624-w · Cited by 155

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

Systemic Microdystrophin Gene Delivery Improves Skeletal Muscle Structure and Function in Old Dystrophic mdx Mice

Gregorevic, P., Blankinship, M. J., Allen, J. M., & Chamberlain, J. S. (2008) · Molecular Therapy · doi:10.1038/mt.2008.28 · Cited by 120

Smad7 gene delivery prevents muscle wasting associated with cancer cachexia in mice

Winbanks, C. E., Murphy, K. T., Bernardo, B. C., Qian, H., Liu, Y., Sepulveda, P. V., Beyer, C., Hagg, A., Thomson, R. E., Chen, J. L., et al. (2016) · Science Translational Medicine · doi:10.1126/scitranslmed.aac4976 · Cited by 95

Gene Therapy of mdx Mice With Large Truncated Dystrophins Generated by Recombination Using rAAV6

Odom, G. L., Gregorevic, P., Allen, J. M., & Chamberlain, J. S. (2011) · Molecular Therapy · doi:10.1038/mt.2010.205 · Cited by 95

Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic Approaches

Shimizu-Motohashi, Y., Komaki, H., Motohashi, N., Takeda, S., Yokota, T., & Aoki, Y. (2019) · Journal of Personalized Medicine · doi:10.3390/jpm9010001 · Cited by 82

Transient Immunomodulation Allows Repeated Injections of AAV1 and Correction of Muscular Dystrophy in Multiple Muscles

Lorain, S., Gross, D.-A., Goyenvalle, A., Danos, O., Davoust, J., & Garcia, L. (2008) · Molecular Therapy · doi:10.1038/sj.mt.6300377 · Cited by 62

A CEP290 C-Terminal Domain Complements the Mutant CEP290 of Rd16 Mice In Trans and Rescues Retinal Degeneration

Mookherjee, S., Chen, H. Y., Isgrig, K., Yu, W., Hiriyanna, S., Levron, R., Li, T., Colosi, P., Chien, W., Swaroop, A., et al. (2018) · Cell Reports · doi:10.1016/j.celrep.2018.09.043 · Cited by 41

The AAV-mediated and RNA-guided CRISPR/Cas9 system for gene therapy of DMD and BMD

Wang, J.-Z., Wu, P., Shi, Z.-M., Xu, Y.-L., & Liu, Z.-J. (2017) · Brain and Development · doi:10.1016/j.braindev.2017.03.024 · Cited by 41

Evaluation of vascular delivery methodologies to enhance rAAV6-mediated gene transfer to canine striated musculature

Gregorevic, P., Schultz, B. R., Allen, J. M., Halldorson, J. B., Blankinship, M. J., Meznarich, N. A., Kuhr, C. S., Doremus, C., Finn, E., Liggitt, D., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2009.116 · Cited by 40

C-reactive protein (CRP) is essential for efficient systemic transduction of recombinant adeno-associated virus vector 1 (rAAV-1) and rAAV-6 in mice

Denard, J., Marolleau, B., Jenny, C., Rao, T. N., Fehling, H. J., Voit, T., & Svinartchouk, F. (2013) · Journal of Virology · doi:10.1128/jvi.01813-13 · Cited by 31