Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy

• Main Authors: Liubov V. Gushchina (Author), Tatyana A. Vetter (Author), Emma C. Frair (Author), Adrienne J. Bradley (Author), Kelly M. Grounds (Author), Jacob W. Lay (Author), Nianyuan Huang (Author), Aisha Suhaiba (Author), Frederick J. Schnell (Author), Gunnar Hanson (Author), Tabatha R. Simmons (Author), Nicolas Wein (Author), Kevin M. Flanigan (Author)
• Format: Book
• Published: Elsevier, 2022-12-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Duchenne muscular dystrophy (DMD) is a devastating muscle-wasting disease that arises due to the loss of dystrophin expression, leading to progressive loss of motor and cardiorespiratory function. Four exon-skipping approaches using antisense phosphorodiamidate morpholino oligomers (PMOs) have been...