AAV-mediated editing of PMP22 rescues Charcot-Marie-Tooth disease type 1A features in patient-derived iPS Schwann cells

BACKGROUND: Charcot-Marie-Tooth disease type 1A (CMT1A) is one of the most common hereditary peripheral neuropathies caused by duplication of 1.5 Mb genome region including PMP22 gene. We aimed to correct the duplication in human CMT1A patient-derived iPS cells (CMT1A-iPSCs) by genome editing and in...

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Detalles Bibliográficos
Autores principales: Yoshioka, Yuki, Taniguchi, Juliana Bosso, Homma, Hidenori, Tamura, Takuya, Fujita, Kyota, Inotsume, Maiko, Tagawa, Kazuhiko, Misawa, Kazuharu, Matsumoto, Naomichi, Nakagawa, Masanori, Inoue, Haruhisa, Tanaka, Hikari, Okazawa, Hitoshi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10684506/
https://www.ncbi.nlm.nih.gov/pubmed/38017287
http://dx.doi.org/10.1038/s43856-023-00400-y

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10684506/
https://www.ncbi.nlm.nih.gov/pubmed/38017287
http://dx.doi.org/10.1038/s43856-023-00400-y

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