AAV-Mediated Gene Supplementation Therapy in Achromatopsia Type 2: Preclinical Data on Therapeutic Time Window and Long-Term Effects

Achromatopsia type 2 (ACHM2) is a severe, inherited eye disease caused by mutations in the CNGA3 gene encoding the α subunit of the cone photoreceptor cyclic nucleotide-gated (CNG) channel. Patients suffer from strongly impaired daylight vision, photophobia, nystagmus, and lack of color discriminati...

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Detalles Bibliográficos
Autores principales: Mühlfriedel, Regine, Tanimoto, Naoyuki, Schön, Christian, Sothilingam, Vithiyanjali, Garcia Garrido, Marina, Beck, Susanne C., Huber, Gesine, Biel, Martin, Seeliger, Mathias W., Michalakis, Stylianos
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2017
Materias:
Neuroscience
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5442229/
https://www.ncbi.nlm.nih.gov/pubmed/28596720
http://dx.doi.org/10.3389/fnins.2017.00292

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