Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis

The aim of this study was the evaluation of the safety and efficacy of unilateral subretinal injection of the adeno-associated vector (AAV) serotypes 2 and 4 (AAV2/4) RPE65-RPE65 vector in patients with Leber congenital amaurosis (LCA) associated with RPE65 gene deficiency. We evaluated ocular and g...

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Опубликовано в: :Mol Ther
Главные авторы: Le Meur, Guylène, Lebranchu, Pierre, Billaud, Fanny, Adjali, Oumeya, Schmitt, Sébastien, Bézieau, Stéphane, Péréon, Yann, Valabregue, Romain, Ivan, Catherine, Darmon, Christophe, Moullier, Philippe, Rolling, Fabienne, Weber, Michel
Формат: Artigo
Язык:Inglês
Опубликовано: American Society of Gene & Cell Therapy 2018
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Original Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC5763029/
https://ncbi.nlm.nih.gov/pubmed/29033008
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.09.014
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