Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice

Genome editing therapy for Duchenne muscular dystrophy (DMD) holds great promise, however, one major obstacle is delivery of the CRISPR-Cas9/sgRNA system to skeletal muscle tissues. In general, AAV vectors are used for in vivo delivery, but AAV injections cannot be repeated because of neutralization...

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Detalles Bibliográficos
Autores principales: Kenjo, Eriya, Hozumi, Hiroyuki, Makita, Yukimasa, Iwabuchi, Kumiko A., Fujimoto, Naoko, Matsumoto, Satoru, Kimura, Maya, Amano, Yuichiro, Ifuku, Masataka, Naoe, Youichi, Inukai, Naoto, Hotta, Akitsu
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8654819/
https://www.ncbi.nlm.nih.gov/pubmed/34880218
http://dx.doi.org/10.1038/s41467-021-26714-w

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