Engineered AAV capsids mediate transduction of murine neurofibroma and sciatic nerve

Genetic diseases such as Neurofibromatosis type 1 (NF1) and Charcot-Marie Tooth disease involve Schwann cells (SCs) associated with peripheral nerves. Gene therapy using adeno-associated virus (AAV) vector mediated gene delivery is a promising strategy to treat these diseases. However, AAV-mediated...

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Main Authors: Abou Haidar, Edwina, Prabhakar, Shilpa, Cheah, Pike See, Hanlon, Killian S., Espinoza, Paula, Crain, Adam V., Patel, Nikita, Radcliff, Greta W., Cheng, Ming, Hernández, Iván Coto, Minderler, Steven, de la Cruz, Demitri, Ng, Carrie, da Hora, Cintia Carla, Charest, Alain, Stemmer-Rachamimov, Anat, Jowett, Nate, Breakefield, Xandra O., Maguire, Casey A.
Format: Article
Language:en
Published: Springer Nature 2025
Online Access:http://psasir.upm.edu.my/id/eprint/120712/1/120712.pdf
http://psasir.upm.edu.my/id/eprint/120712/
https://www.nature.com/articles/s41434-025-00542-9?error=cookies_not_supported&code=b32cb9ec-f337-4c46-868a-ee66fac16f7c
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