期刊
DEVELOPMENTAL DISABILITIES RESEARCH REVIEWS
卷 16, 期 2, 页码 219-229出版社
WILEY
DOI: 10.1002/ddrr.109
关键词
mitochondrial diseases; gene therapy; heteroplasmy; oxidative phosphorylation defects
资金
- PHS [EY10804, NS041777, CA85700]
- Parkinson Disease Foundation
- Muscular Dystrophy Association
- United Mitochondria Disease Foundation
- NATIONAL CANCER INSTITUTE [R01CA085700] Funding Source: NIH RePORTER
- NATIONAL EYE INSTITUTE [R01EY010804] Funding Source: NIH RePORTER
- NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE [R01NS041777, R56NS041777] Funding Source: NIH RePORTER
Mitochondrial diseases are very heterogeneous and can affect different tissues and organs. Moreover, they can be caused by genetic defects in either nuclear or mitochondrial DNA as well as by environmental factors. All of these factors have made the development of therapies difficult. In this review article, we will discuss emerging approaches to the therapy of mitochondrial disorders, some of which are targeted to specific conditions whereas others may be applicable to a more diverse group of patients. (C) 2010 Wiley-Liss, Inc. Day Disabil Res Rev 2010;16:219-229.
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