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

Yuki Yoshioka Juliana Bosso Taniguchi Hidenori Homma Takuya Tamura Kyota FujitaMaiko InotsumeKazuhiko TagawaKazuharu MisawaNaomichi MatsumotoMasanori NakagawaHaruhisa InoueHikari TanakaHitoshi Okazawa

Published in: Communications medicine (2023)

In vivo transfer of AAV2-hSaCas9-gRNAedit to peripheral nerves could be a potential therapeutic modality for CMT1A patient after careful examinations of toxicity including off-target mutations.

Keyphrases

induced apoptosis
gene therapy
crispr cas
cell cycle arrest
oxidative stress
case report
mouse model
cell death
peripheral nerve
signaling pathway
pi k akt