Gene responsible for Duchenne muscular dystrophy can be repaired.Washington, Apr 16 (ANI): It is possible to repair the defective gene responsible for Duchenne muscular dystrophy Duchenne muscular dystrophy (DMD)
The most severe form of muscular dystrophy, DMD usually affects young boys and causes progressive muscle weakness, usually beginning in the legs. , claim researchers from Universite Laval's Faculty of Medicine and the CHUQ CHUQ Centre Hospitalier Universitaire de Quebec (Canada) Research Center.
The team, led by Professor Jacques P. Tremblay, is presenting its new therapeutic approach in an article published in the online version of the scientific journal Gene Therapy.
Duchenne muscular dystrophy is a hereditary disease. It causes progressive muscle degeneration that begins in early childhood and causes death by age 25 in most people afflicted. The disease is caused by mutations that affect a protein called "dystrophin dys·tro·phin
A structural protein found in small amounts in normal muscle but absent or present in abnormal amounts in individuals with muscular dystrophy. ." The mutations alter the normal nucleotide sequences of this protein's gene and stop its synthesis.
Professor Tremblay's team partnered with Cellectis, a French firm specializing in genome engineering, in order to design enzymes-called meganucleases-with the ability to correct the dystrophin gene. During in vitro testing, the researchers inserted genes coding for a variety of meganucleases into human muscle cells.
They repeated the experiment in vivo with mice carrying the mutation that causes the illness. Both series of testing showed that the meganucleases can lead to a restoration of the normal nucleotide sequences of the dystrophin gene and its expression in muscle cells. (ANI)
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