Type Of Media：患者啓発Web媒体
Publication/Magazine/Media：RareS
Successful treatment of a mouse model of Duchenne muscular dystrophy (DMD) using mRNA medicine.
Summary:
The research team, including Dr. Keishi Iitaka (Professor, Tokyo University of Science), in collaboration with the National Center of Neurology and Psychiatry (NCNP) and Sapporo Medical University, has successfully treated Duchenne Muscular Dystrophy (DMD) using an mRNA medicine. DMD is a genetic disease that causes a deficiency of dystrophin, which is necessary for the maintenance of muscle structure, and is currently treated mainly by symptomatic therapy. Previous exon-skipping therapies have been limited to specific genetic mutations.
In this study, we used PGC-1α, a protein that enhances mitochondrial activity, and widely administered mRNA to muscle tissue through nanomicellar mRNA carriers. In the experiment, administration of PGC-1α mRNA to DMD model mice resulted in suppression of exercise-induced muscle weakness and significant reduction in muscle tissue damage. Gene expression analysis showed increased expression of genes related to mitochondrial activity and muscle activity, suggesting that PGC-1α mRNA contributed to the improvement of muscle symptoms. This study demonstrates the possibility of a new therapeutic approach using mRNA and is expected to be a major step toward expanding treatment options for intractable diseases.
http://raresnet.com/250403-01/
