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...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:CRISPR J
Egile Nagusiak: 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.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: Mary Ann Liebert, Inc. 2018
Gaiak:
Research Articles
Sarrera elektronikoa: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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