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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Bibliographic Details
Published in:Hum Gene Ther
Main Authors: 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
Language:Inglês
Published: Mary Ann Liebert, Inc. 2016
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Research Articles
Online Access: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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