Nucleic Acid Therapeutics for Neurological Diseases
RNA Therapeutics Institute; Department of Neurology
Biochemistry, Biophysics, and Structural Biology | Genetics and Genomics | Nervous System Diseases | Nucleic Acids, Nucleotides, and Nucleosides | Therapeutics
Oligonucleotide therapeutics are emerging as a promising class of therapeutics in various areas of human health. In the long run, oligonucleotides are likely to become a major class of therapeutics, on par with small molecules and biologics. Initially, oligonucleotides were predominantly used for modulation of gene expression in the liver, in which robust clinical efficacy has been demonstrated with both antisense [1, 2] and siRNA  compounds reaching approval.
DOI of Published Version
Watts JK, Brown RH, Khvorova A. Nucleic Acid Therapeutics for Neurological Diseases. Neurotherapeutics. 2019 Apr;16(2):245-247. doi: 10.1007/s13311-019-00736-1. PMID: 31037650; PMCID: PMC6554381. Link to article on publisher's site
Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics
Watts JK, Brown RH, Khvorova A. (2019). Nucleic Acid Therapeutics for Neurological Diseases. RNA Therapeutics Institute Publications. https://doi.org/10.1007/s13311-019-00736-1. Retrieved from https://escholarship.umassmed.edu/rti_pubs/66