Scientists manage to edit heart muscle cells with mutations that lead to the development of Duchenne muscular dystrophy. Using the CRISPR-Cas9 system, experts “thrown out” mutant regions from mRNA, and grew a working heart muscle from “corrected” cells..

The DMD gene is considered the largest human genome. He is engaged in encoding dystrophin, which ensures the connection of muscle fibers with the matrix. If the synthesis of this protein is disturbed, then Duchenne myodystrophy begins to develop in the gene.. In addition, patients stop walking, and eventually die in 20-30 years..

Researchers from the American Southwestern Medical Center at the University of Texas managed to implement "

Almost 3 thousand mutations in the DMD gene that determine the development of Duchenne muscular dystrophy and are grouped into mutagenesis, concentrated in 12 exons.

With the help of the system, scientists have found guide RNAs to make reserves in all these zones.. This means that experts have created an exon skipping kit that is suitable for almost all patients..

Scientists conducted research on mice, and then on human heart dream cells. It turned out that the CRISPR system and exon skipping effectively restores the expression of dystrophin in cell culture..

During the tests, scientists have grown an artificial heart muscle. It turned out that the “edited” muscle contracted normally. Only 30-50 cells are enough to maintain contractile function.

Experts called the new discovery " It will be used to edit cells with further implantation in the heart muscle..

aspect. net.