Prime editing optimized RTT permits the correction of the c.8713C>T mutation in DMD gene

• Main Authors: Cedric Happi Mbakam (Author), Joel Rousseau (Author), Yaoyao Lu (Author), Anne Bigot (Author), Kamel Mamchaoui (Author), Vincent Mouly (Author), Jacques P. Tremblay (Author)
• Formato: Libro
• Publicado: Elsevier, 2022-12-01T00:00:00Z.
• Subjects: article
• Acceso en liña: Connect to this object online.

Duchenne muscular dystrophy is a severe debilitating genetic disease caused by different mutations in the DMD gene leading to the absence of dystrophin protein under the sarcolemma. We used CRISPR-Cas9 prime editing technology for correction of the c.8713C>T mutation in the DMD gene and tested di...