Efficient and Precise CRISPR/Cas9-Mediated MECP2 Modifications in Human-Induced Pluripotent Stem Cells

Patients with Rett syndrome (RTT) have severe mental and physical disabilities. The majority of RTT patients carry a heterozygous mutation in methyl-CpG binding protein 2 (MECP2), an X-linked gene encoding an epigenetic factor crucial for normal nerve cell function. No curative therapy for RTT syndr...

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Detalles Bibliográficos
Autores principales: Le, Thi Thanh Huong, Tran, Ngoc Tung, Dao, Thi Mai Lan, Nguyen, Dinh Dung, Do, Huy Duong, Ha, Thi Lien, Kühn, Ralf, Nguyen, Thanh Liem, Rajewsky, Klaus, Chu, Van Trung
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2019
Materias:
Genetics
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6614930/
https://www.ncbi.nlm.nih.gov/pubmed/31333716
http://dx.doi.org/10.3389/fgene.2019.00625

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