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Short-Term Outcomes of the First in Vivo Gene Therapy for RPE65-Mediated Retinitis Pigmentosa

Here, we report early treatment outcomes of gene therapy for early onset retinitis pigmentosa (RP) (Leber congenital amaurosis) associated with biallelic RPE65 mutation in a 30-year-old female patient. Initially, her visual acuity (VA) was 20/200, and her visual field (VF) was severely constricted t...

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Detalles Bibliográficos
Autores principales: Kwak, Jay Jiyong, Kim, Hae Rang, Byeon, Suk Ho
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Yonsei University College of Medicine 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9226827/
https://www.ncbi.nlm.nih.gov/pubmed/35748082
http://dx.doi.org/10.3349/ymj.2022.63.7.701