Gene Editing Restores Dystrophin Function in Stem Cells From Patients With Severe Muscle Disorder
August 30, 2023| |
Researchers from Kyoto University used gene editing to restore dystrophin protein function in stem cells that originated from Duchenne muscular dystrophy (DMD) patients. This study is a promising approach for the treatment of DMD and other genetic disorders that need extensive deletions.
Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder that is caused by mutations that affect the dystrophin gene. This illness is considered the most frequent and severe kind of muscular dystrophy, and there are no curative treatments for it at the moment.
To address this issue, scientists from Kyoto University used dual CRISPR-Cas3 to remove up to 340 kilobases of the dystrophin gene. This procedure allowed cells to skip damaged or misaligned portions of the genetic code to restore dystrophin protein. "Our dual-Cas3 system might apply to future gene therapies once we're able to deliver the dual-Cas3 components in vivo to skeletal muscle tissues safely and efficiently," said Akitsu Hotta, the senior author of the study.
Read the journal article on Stem Cell Reports for more information.
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