Two Medical Breakthroughs for Spinal Muscular Atrophy Treatment
The antisense oligonucleotides method
A peculiar genetic approach makes this method work. The survival motor neuron 1 or SMN1 is a causative gene that has an echo and a second gene called SMN2, which is near chromosome 5 is in reverse orientation. This situation is similar to sickle cell disease. When the two copies of SMN2 has a glitch, it gets disabled. As a result, it can only produce a small amount of the SMN protein, which is needed by motor neurons. The antisense method fixes the problem by using a fragment of nucleic acid that is like DNA. It binds to the echo gene and frees it to directly synthesize the needed protein. It works to some extent since there are trials done that have had positive results.
Nusinersen (Spinraza) is an FDA approved antisense drug released in 2016. The development of the drug was covered by DNA Science and described one family’s experience. The New England Journal of Medicine gave an update regarding the findings.
