Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy
The University of Texas Southwestern Medical Center
Abstract
CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations in the dystrophin gene. To correct DMD by skipping mutant dystrophin exons in postnatal muscle tissue in vivo, we used adeno-associated virus-9 (AAV9) to deliver gene-editing components to postnatal mdx mice, a model of DMD. Different modes of AAV9 delivery were systematically tested, including intraperitoneal at postnatal day 1 (P1), intramuscular at P12, and retro-orbital at P18. Each of these methods restored dystrophin protein expression in cardiac and skeletal muscle to varying degrees, and expression increased from 3 to 12 weeks after…
Citation impact
- FWCI
- 59.48
- Percentile
- 100%
- References
- 34
Authors
10- CLChengzu LongCorresponding
The University of Texas Southwestern Medical Center
- LALeonela AmoasiiCorresponding
The University of Texas Southwestern Medical Center
- AAAlex A. Mireault
The University of Texas Southwestern Medical Center
- JMJohn McAnally
The University of Texas Southwestern Medical Center
- HLHui Li
The University of Texas Southwestern Medical Center
Topics & keywords
- Dystrophin
- Muscular dystrophy
- Genome
- Biology
- Duchenne muscular dystrophy
- Genetics
- Genome editing
- Utrophin
- Good health and well-being