Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice

Michela Alessandra Denti, Tania Incitti, Olga Sthandier, Carmine Nicoletti, Fernanda Gabriella De Angelis, Emanuele Rizzuto, Alberto Auricchio, Antonio Musarò, Irene Bozzoni

Research output: Contribution to journalArticlepeer-review

56 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)601-608
Number of pages8
JournalHuman gene therapy
Volume19
Issue number6
DOIs
StatePublished - Jun 1 2008
Externally publishedYes

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