期刊
MOLECULAR GENETICS AND METABOLISM
卷 107, 期 3, 页码 617-619出版社
ACADEMIC PRESS INC ELSEVIER SCIENCE
DOI: 10.1016/j.ymgme.2012.09.019
关键词
Methylmalonic acidemia; MMA; Gene therapy; Adeno-associated virus; AAV; Organic acidemia
资金
- Intramural NIH HHS [Z99 HG999999, ZIA HG200318-09] Funding Source: Medline
We demonstrate that human methylmalonyl-CoA mutase (MUT), delivered using an AAV serotype 8 vector, rescues the lethal phenotype displayed by mice with MMA and provides long-term phenotypic correction. In addition to defining a lower limit of effective dosing, our studies establish that neither a species barrier to mitochondrial processing nor an apparent immune response to MUT limits the murine model as an experimental platform to test the efficacy of human gene therapy vectors for MMA. Published by Elsevier Inc.
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