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...

पूर्ण विवरण

में बचाया:
ग्रंथसूची विवरण
में प्रकाशित:Mol Ther
मुख्य लेखकों: 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
स्वरूप: Artigo
भाषा:Inglês
प्रकाशित: American Society of Gene & Cell Therapy 2018
विषय:
Original Article
ऑनलाइन पहुंच: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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