Spinal Muscular Atrophy (SMA) and mRNA-based therapy against it

Abstract

Spinal Muscular Atrophy (SMA), is a genetic disease caused by the loss of nerve cells
called motor neurons. It occurs as a result of the inability to produce the SMN protein,
which is a motor neuron protein. Currently, no treatment has been found for the
complete recovery of people with SMA. However, the symptoms of the disease can be
reduced through medications. The main problem, the inability to produce the SMN
protein, can be achieved by using mRNA-based therapy. The mRNA-based therapy
basically contains the code for the protein we want to be produced, and thus that protein
is produced by the body. This is how the missing protein that causes SMA can be rescued.