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Article

Effective delivery of large genes to the retina by dual AAV vectors.

EMBO molecular medicine - 1 Feb 2014

Trapani Ivana, Colella Pasqualina, Sommella Andrea, Iodice Carolina, Cesi Giulia, de Simone Sonia, Marrocco Elena, Rossi Settimio, Giunti Massimo, Palfi Arpad, Farrar Gwyneth J, Polishchuk Roman, Auricchio Alberto

Abstract excerpt

Retinal gene therapy with adeno-associated viral (AAV) vectors is safe and effective in humans. However, AAV's limited cargo capacity prevents its application to therapies of inherited retinal diseases due to mutations of genes over 5 kb, like Stargardt's disease (STGD) and Usher syndrome type IB (USH1B). Previous methods based on 'forced' packaging of large genes into AAV capsids may not be easily translated to...

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Identifiers

PMID: 24150896
DOI: 10.1002/emmm.201302948
PMCID: PMC3927955

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