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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 |
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| Главные авторы: | , , , , , , , , , , , , |
| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
American Society of Gene & Cell Therapy
2018
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| Предметы: | |
| 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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