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.

Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice / Michela Alessandra, Denti; Incitti, Tania; Sthandier, Olga Elena; Nicoletti, Carmine; F. G., De Angelis; Rizzuto, Emanuele; Alberto, Auricchio; Musaro', Antonio; Bozzoni, Irene. - In: HUMAN GENE THERAPY. - ISSN 1043-0342. - STAMPA. - 19:6(2008), pp. 601-608. [10.1089/hum.2008.012]

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

INCITTI, TANIA;STHANDIER, Olga Elena;NICOLETTI, CARMINE;RIZZUTO, EMANUELE;MUSARO', Antonio;BOZZONI, Irene
2008

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.
2008
01 Pubblicazione su rivista::01a Articolo in rivista
Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice / Michela Alessandra, Denti; Incitti, Tania; Sthandier, Olga Elena; Nicoletti, Carmine; F. G., De Angelis; Rizzuto, Emanuele; Alberto, Auricchio; Musaro', Antonio; Bozzoni, Irene. - In: HUMAN GENE THERAPY. - ISSN 1043-0342. - STAMPA. - 19:6(2008), pp. 601-608. [10.1089/hum.2008.012]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11573/357853
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