A new hope for curing Amyotrophic lateral sclerosis
The “Téléthon” occurs this weekend everywhere in France. Thanks to this annual event, major research programs have made great advances to better know and cure devastating genetic diseases like Duchenne Muscular Dystrophy or Spinal Muscular Atrophy.
As an example of the excellence of this research, Dr. Maria Grazia Biferi (Centre of Research in Myology (CRM), French Institute of Myology, Paris), Dr. Martine Barkats and their colleagues successfully designed, conceived and optimized a gene therapy protocol for ALS (Amyotrophic Lateral Sclerosis). This disease is the most common adult-onset disorder affecting spinal motor neurons and is characterized by severe paralysis and death within 3–5 years after diagnosis. Unfortunately, no treatment is currently available to offer a robust survival benefit or at least a significant retardation of symptom onset.
In this article, They successfully reported an innovative molecular strategy for inducing substantial, widespread, and sustained reduction of mutant human SOD1, which is accumulated in the motor neurons of 12% of fALS cases. Adeno-associated virus serotype rh10 vectors (AAV10) were used to mediate exon skipping of hSOD1, reducing then its production and lethal accumulation in motor neurons. This treatment increased the survival of ALS mice, and prevented weight loss and the decline of neuromuscular function.
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