Abstract

Antisense oligonucleotides, which are designed to bind to and modulate the function of protein coding and noncoding RNAs in a sequence specific manner, are being developed for the treatment of a broad range of diseases. An emerging area for antisense based drugs is for the treatment of neurological diseases. There is one approved antisense drug, nusinersen, for the treatment of spinal muscular atrophy and at least fourteen antisense drugs currently in clinical trials for the treatment of multiple diseases affecting the central nervous system, with additional antisense drugs are in late-stage research. We have demonstrated that antisense oligonucleotides distribute broadly in CNS tissues following intrathecal delivery and well tolerated can be identified through an extensive screening process. I will provide an update on what is being learned preclinically and clinically regarding the application of antisense technology as potential therapies for neurological diseases.