Persistence of non-viral vector mediated RPE65 expression: case for viability as a gene transfer therapy for RPE-based diseases

Mutations in the retinal pigment epithelium (RPE) gene RPE65 are associated with multiple blinding diseases including Leber’s Congenital Amaurosis (LCA). Our goal has been to develop persistent, effective non-viral genetic therapies to treat this condition. Using precisely engineered DNA vectors and...

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Autores principales: Koirala, Adarsha, Conley, Shannon M., Makkia, Rasha, Liu, Zhao, Cooper, Mark J, Sparrow, Janet R., Naash, Muna I.
Formato: Artigo
Lenguaje:Inglês
Publicado: 2013
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3858392/
https://ncbi.nlm.nih.gov/pubmed/24035979
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jconrel.2013.08.299
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