CRISPR-Cas9-Mediated Correction of the 1.02 kb Common Deletion in CLN3 in Induced Pluripotent Stem Cells from Patients with Batten Disease

Juvenile neuronal ceroid lipofuscinosis (Batten disease) is a rare progressive neurodegenerative disorder caused by mutations in CLN3. Patients present with early-onset retinal degeneration, followed by epilepsy, progressive motor deficits, cognitive decline, and premature death. Approximately 85% o...

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Detalhes bibliográficos
Publicado no:CRISPR J
Main Authors: Burnight, Erin R., Bohrer, Laura R., Giacalone, Joseph C., Klaahsen, Darcey L., Daggett, Heather T., East, Jade S., Madumba, Robert A., Worthington, Kristan S., Mullins, Robert F., Stone, Edwin M., Tucker, Budd A., Wiley, Luke A.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2018
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6319325/
https://ncbi.nlm.nih.gov/pubmed/31021193
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/crispr.2017.0015
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