mRNA-mediated delivery of gene editing tools to human primary muscle stem cells

Muscular dystrophies are approximately 50 devastating, untreatable monogenic diseases leading to progressive muscle degeneration and atrophy. Gene correction of transplantable cells using CRISPR/Cas9-based tools is a realistic scenario for autologous cell replacement therapies to restore organ funct...

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Detalles Bibliográficos
Autores principales: Stadelmann, Christian, Di Francescantonio, Silvia, Marg, Andreas, Müthel, Stefanie, Spuler, Simone, Escobar, Helena
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8931293/
https://www.ncbi.nlm.nih.gov/pubmed/35356683
http://dx.doi.org/10.1016/j.omtn.2022.02.016

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8931293/
https://www.ncbi.nlm.nih.gov/pubmed/35356683
http://dx.doi.org/10.1016/j.omtn.2022.02.016

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