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Wang Z, Tapscott SJ, Chamberlain JS, et al. Immunity and AAV-Mediated Gene Therapy for Muscular Dystrophies in Large Animal Models and Human Trials. Front Microbiol. 2011;2:201doi: 10.3389/fmicb.2011.00201.
Wang, Z., Tapscott, S. J., Chamberlain, J. S., & Storb, R. (2011). Immunity and AAV-Mediated Gene Therapy for Muscular Dystrophies in Large Animal Models and Human Trials. Frontiers in microbiology, 2201. https://doi.org/10.3389/fmicb.2011.00201
Wang, Zejing, et al. "Immunity and AAV-Mediated Gene Therapy for Muscular Dystrophies in Large Animal Models and Human Trials." Frontiers in microbiology vol. 2 (2011): 201. doi: https://doi.org/10.3389/fmicb.2011.00201
Wang Z, Tapscott SJ, Chamberlain JS, Storb R. Immunity and AAV-Mediated Gene Therapy for Muscular Dystrophies in Large Animal Models and Human Trials. Front Microbiol. 2011 Sep 26;2:201. doi: 10.3389/fmicb.2011.00201. eCollection 2011. PMID: 21980317; PMCID: PMC3180173.
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Front Microbiol. 2011 Sep 26;2:201. doi: 10.3389/fmicb.2011.00201. eCollection 2011.

Immunity and AAV-Mediated Gene Therapy for Muscular Dystrophies in Large Animal Models and Human Trials.

Frontiers in microbiology

Zejing Wang, Stephen J Tapscott, Jeffrey S Chamberlain, Rainer Storb

Affiliations

  1. Division of Clinical Research, Fred Hutchinson Cancer Research Center Seattle, WA, USA.

PMID: 21980317 PMCID: PMC3180173 DOI: 10.3389/fmicb.2011.00201

Abstract

Adeno-associated viral (AAV) vector-mediated gene replacement for the treatment of muscular dystrophy represents a promising therapeutic strategy in modern medicine. One major obstacle in using AAV vectors for in vivo gene delivery is the development of host immune responses to the viral capsid protein and transgene products as evidenced in animal models and human trials for a range of genetic diseases. Here, we review immunity against AAV vector and transgene in the context of gene delivery specific to muscles for treating muscular dystrophies and non-muscle diseases in large animal models and human trials, factors that influence the intensity of the immune responses, and immune modulatory strategies to prevent unwanted immune responses and induce tolerance to the vector and therapeutic gene for a successful gene therapy.

Keywords: AAV; immune modulation; immunity; muscular dystrophy; review

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