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Gene Therapy of Inherited Retinopathies: A Long and Successful Road from Viral Vectors to Patients

26 Jun 2012

Abstract excerpt

Inherited retinopathies (IRs) are common and untreatable blinding conditions inherited mostly as monogenic due to mutations in genes expressed in retinal photoreceptors (PRs) and in retinal pigment epithelium (RPE). Over the last two decades, the retina has emerged as one of the most favorable target tissues for gene therapy given its small size and its enclosed and immune-privileged environment. Different types...

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PMID: 22734691
DOI: 10.1089/hum.2012.123

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