CRISPR/Cas9 Genome Editing in LGMD2A/R1 Patient-Derived Induced Pluripotent Stem and Skeletal Muscle Progenitor Cells

• Main Authors: Lampros Mavrommatis (Author), Abdul Zaben (Author), Urs Kindler (Author), Marie-Cécile Kienitz (Author), Julienne Dietz (Author), Hyun-Woo Jeong (Author), Pierre Böhme (Author), Beate Brand-Saberi (Author), Matthias Vorgerd (Author), Holm Zaehres (Author)
• Format: Book
• Published: Hindawi Limited, 2023-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Large numbers of Calpain 3 (CAPN3) mutations cause recessive forms of limb-girdle muscular dystrophy (LGMD2A/LGMDR1) with selective atrophy of the proximal limb muscles. We have generated induced pluripotent stem cells (iPSC) from a patient with two mutations in exon 3 and exon 4 at the calpain 3 lo...