Outcomes of progranulin gene therapy in the retina are dependent on time and route of delivery

Neuronal ceroid lipofuscinosis (NCL) is a family of neurodegenerative diseases caused by mutations to genes related to lysosomal function. One variant, CNL11, is caused by mutations to the gene encoding the protein progranulin, which regulates neuronal lysosomal function. Absence of progranulin caus...

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Detalles Bibliográficos
Autores principales: Zin, Emilia A., Han, Daisy, Tran, Jennifer, Morisson-Welch, Nikolas, Visel, Meike, Kuronen, Mervi, Flannery, John G.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8390452/
https://www.ncbi.nlm.nih.gov/pubmed/34485593
http://dx.doi.org/10.1016/j.omtm.2021.05.009

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8390452/
https://www.ncbi.nlm.nih.gov/pubmed/34485593
http://dx.doi.org/10.1016/j.omtm.2021.05.009

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