Review on gene replacement therapy for spinal muscular atrophy

Abstract

Spinal muscular atrophy (SMA) is initiated by the deletion or malfunction of the gene that encodes survival motor neuron 1 (SMN1) and this leads to muscular atrophy. It is one of the most prevalent hereditary that causes infant mortality. Eventually, various genetic interventions and approaches, including gene replacement, non-gene replacement, and gene editing have been explored up to this point as potential treatment options for spinal muscular atrophy and other neuromuscular illnesses. Whereas gene replacement therapy is most often claimed to treat infants and toddlers with SMA type I. When treating the underlying cause of this disease, researchers have discovered the greatest benefit to motor neurons early on. Here, I have reviewed the enormous advances made in genetically mediated therapeutics for SMA, with an emphasis on gene replacement therapy and the outcome of this therapy was found to be more efficacious than other treatments in terms of motor millstone result.