Ayşenur Saygılı1, Berna Özdem2, Oytun Erbaş3

1Department of Bioengineering, Karamanoğlu Mehmetbey University, Karaman, Türkiye
2Department of Medical Biology and Genetics, Inönü University, Malatya, Türkiye
3Institute of Experimental Medicine, Gebze-Kocaeli, Türkiye

Keywords: Antisense oligonucleotides, gene therapy, ribonucleic acid, spinal muscular atrophy, viral vector.


Spinal muscular atrophy (SMA) is an uncommon neuromuscular disorder characterized by the gradual degeneration and weakening of muscles. The condition often manifests in the pediatric and teenage populations, however, it may also impact adults. It is characterized by the manifestation of muscle weakness and muscle wasting, leading to a range of consequences such as diminished strength, respiratory impairment, and potentially fatal outcomes. In recent times, there has been a notable emergence of novel therapeutic interventions aimed at addressing this particular ailment. Spinal muscular atrophy is caused by low levels of survival motor neuron (SMN) protein resulting from SMN1 gene mutations or deletions. Until 2016, no treatment for SMA was available. Treatment prospects were added with the recent approval of two groundbreaking SMN-targeted therapies using antisense oligonucleotides or virus-mediated gene therapy. These two new therapeutics have a common goal: To increase the production of SMN protein in motor neurons and improve survival by revitalizing motor function. The present review endeavors to go into an in-analysis of various therapy alternatives.

Cite this article as: Saygılı A, Özdem B, Erbaş O. Spinal muscular atrophy and current new generation treatment. D J Tx Sci 2023;8(1-2):27-34. doi: 10.5606/ dsufnjt.2023.10.

Author Contributions

All authors contributed equally to the article.

Data Sharing Statement:
The data that support the findings of this study are available from the corresponding author upon reasonable request.

Conflict of Interest

The authors declared no conflicts of interest with respect to the authorship and/ or publication of this article.

Financial Disclosure

The authors received no financial support for the research and/or authorship of this article.