期刊
FRONTIERS IN MICROBIOLOGY
卷 2, 期 -, 页码 -出版社
FRONTIERS RESEARCH FOUNDATION
DOI: 10.3389/fmicb.2011.00201
关键词
AAV; immunity; muscular dystrophy; immune modulation; review
类别
资金
- NIH [P30-CA15704, R01-AR056949, R01-AR041928, R37-AR40864]
- Career Development Grant
- Muscular Dystrophy Association [MDA 114979]
- NATIONAL INSTITUTE OF ARTHRITIS AND MUSCULOSKELETAL AND SKIN DISEASES [R01AR056949] Funding Source: NIH RePORTER
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.
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