Details of a new German research showing that a drug called valproate (already used to treat epilepsy) could influence the behavior of a gene in patients with debilitating genetic condition have been presented at the European Society of Human Genetics conference in
Spinal muscular atrophy, a relatively common inherited disease was the focus of the study. Instead of replacing a faulty gene, the gene therapy experts found a new approach, persuading the cells to repair by utilizing its own correctional mechanism.
Spinal muscular atrophy (SMA) is the lading cause of death infants.
This is not a cure, but a promise of a cure.
Read more at The Biotech Weblog and the full report at BBC Health News.
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