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

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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Bibliographic Details
Main Authors: Christian Stadelmann, Silvia Di Francescantonio, Andreas Marg, Stefanie Müthel, Simone Spuler, Helena Escobar
Format: Article
Language:English
Published: Elsevier 2022-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: DNA editing
human muscle stem cells
CRISPR/Cas9
gene editing
base editing
muscular dystrophy
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253122000440

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http://www.sciencedirect.com/science/article/pii/S2162253122000440

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