Researchers Use Gene Therapy to Counter Fatal Spinal Muscular Disease

Published By : 02 Nov 2017 | Published By : QYRESEARCH

Spinal muscular atrophy is a neuromuscular disease that is generally considered a death sentence for affected children, as there is no cure and the progression of the disease is certain. Spinal muscular atrophy destroys the nerve cells in a baby’s body in a progressive manner, leading to the babies being unable to move, swallow, and eventually breathe. New research, however, has extended the frontiers with gene therapy, with which they replaced the defective genes in the babies in the first few months of their life. This has the potential to be a revolutionary contribution to spinal muscular atrophy treatment, albeit more widespread studies are required before the treatment becomes mainstream.

15 babies were administered the experimental gene therapy, with some of them now even able to sit and stand on their own. The researchers themselves are the first to admit more research is needed in the field, while even the experimental therapy itself remains unproven in the long term so far. Spinal muscular atrophy has a prevalence rate of about 1 in 10,000 births. The deadliest form of the disease gives patients grave prognostications, with less than 8% surviving to the age of 20 years without requiring artificial breathing.

12 babies in the group were given high doses of the gene, carried to the motor neurons by a virus modified to be unable to cause the disease. 3 were given a low dose as a safety precaution. Promisingly, the ones who received high doses exhibited a corresponding advantage in their improvement. They were able to swallow, sit, stand up, and speak, which already represents a huge leap from where the treatment of spinal muscular atrophy stands at present.

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