New Research In Treatment for Spinal Muscle Atrophy (SMA)
11:43 PM Posted In disease , gene therpay , research , SMA , spinal muscular atrophy Edit This 12 Comments »
Science Translation Medicine has just published groundbreaking research on spinal muscle atrophy (SMA.)
SMA is a set of inherited muscle diseases that cause muscle to continuously degenerate and weaken. The disease requires extensive and ongoing medical care. The disease process usually ends with death. In fact, SMA is one of the most common genetic causes of death in childhood. Sadly, there is no known cure or effective treatment.
The research by University of Sheffield demonstrated a novel gene transfer system that has the promise of efficient therapeutic treatment for SMA patients. I will let you read the research process below, but the approach has the possibility of establishing gene therapy with the use of just a single injection.
It is important to note that test was conducted using animals, but these findings undoubtedly put science that much closer to actual gene therapy treatments for SMA patients.
Research in depth:
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