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

• Main Authors: Christian Stadelmann (Author), Silvia Di Francescantonio (Author), Andreas Marg (Author), Stefanie Müthel (Author), Simone Spuler (Author), Helena Escobar (Author)
• Format: Book
• Published: Elsevier, 2022-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...