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

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 targe...

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Detalhes bibliográficos
Main Authors: Colella, Pasqualina, Auricchio, Alberto
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2012
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3413895/
https://ncbi.nlm.nih.gov/pubmed/22734691
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2012.123
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