Journal
HUMAN GENE THERAPY
Volume 19, Issue 6, Pages 601-608Publisher
MARY ANN LIEBERT INC
DOI: 10.1089/hum.2008.012
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Funding
- Telethon [TGM03Z09, TGM06S01] Funding Source: Medline
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Many mutations and deletions in the dystrophin gene, responsible for Duchenne muscular dystrophy (DMD), can be corrected at the posttranscriptional level by skipping specific exons. Here we show that long-term benefit can be obtained in the dystrophic mouse model through the use of adeno-associated viral vectors expressing antisense sequences: persistent exon skipping, dystrophin rescue, and functional benefit were observed 74 weeks after a single systemic administration. The therapeutic benefit was sufficient to preserve the muscle integrity of mice up to old age. These results indicate a possible long-term gene therapy treatment of the DMD pathology.
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