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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...

詳細記述

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書誌詳細
主要な著者: Colella, Pasqualina, Auricchio, Alberto
フォーマット: Artigo
言語:Inglês
出版事項: Mary Ann Liebert, Inc. 2012
主題:
オンライン・アクセス: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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