Recent Advances in Antisense Oligonucleotide Therapy in Genetic Neuromuscular Diseases

ASO-based therapeutics, being able to modulate disease pathways are potential candidates for the currently incurable genetic neuromuscular diseases. However, current ASOs do not readily cross an intact blood-brain barrier and limit their application via intrathecal injections for central nervous system diseases. Successful ASO development for other neuromuscular diseases that primarily affect peripheral nerves and skeletal muscles will also require efficient delivery methods.

Please explore the biochemical modification of the oligonucleotide. If you are capable, please provide an alternative delivery system. Feel free to leave comments.