Within a month of each other, a German-American and a Chinese-American team published the proof of concept of a gene therapy strategy leading to the expression of a full-length dystrophin in the skeletal and cardiac muscle of mdx mice. This method is based on intelin-mediated protein trans-splicing.
- It uses triple administration via a highly muscle-tropic AAV (AAVMYO, MyoAAV4A) of three fragments of the DMD gene linked to orthogonal pairs of separate intreins.
- It requires lower doses of AAV than those used in clinical trials of microdystrophin gene therapy.
- It significantly improves muscle function in both young and old mdx mice, more effectively than microdystrophin gene therapy.
