Using Patient-Specific Induced Pluripotent Stem Cells and Wild-Type Mice to Develop a Gene Augmentation-Based Strategy to Treat CLN3-Associated Retinal Degeneration

Juvenile neuronal ceroid lipofuscinosis (JNCL) is a childhood neurodegenerative disease with early-onset, severe central vision loss. Affected children develop seizures and CNS degeneration accompanied by severe motor and cognitive deficits. There is no cure for JNCL, and patients usually die during...

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Publicat a:Hum Gene Ther
Autors principals: Wiley, Luke A., Burnight, Erin R., Drack, Arlene V., Banach, Bailey B., Ochoa, Dalyz, Cranston, Cathryn M., Madumba, Robert A., East, Jade S., Mullins, Robert F., Stone, Edwin M., Tucker, Budd A.
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc. 2016
Matèries:
Research Articles
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5035933/
https://ncbi.nlm.nih.gov/pubmed/27400765
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2016.049
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