Long-Term Structural Outcomes of Late-Stage RPE65 Gene Therapy

The form of hereditary childhood blindness Leber congenital amaurosis (LCA) caused by biallelic RPE65 mutations is considered treatable with a gene therapy product approved in the US and Europe. The resulting vision improvement is well accepted, but long-term outcomes on the natural history of retin...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Gardiner, Kristin L., Cideciyan, Artur V., Swider, Malgorzata, Dufour, Valérie L., Sumaroka, Alexander, Komáromy, András M., Hauswirth, William W., Iwabe, Simone, Jacobson, Samuel G., Beltran, William A., Aguirre, Gustavo D.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2020
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6951840/
https://ncbi.nlm.nih.gov/pubmed/31604676
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.08.013
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