RNA Therapeutics Institute Publications

Title

Nucleic Acid Therapeutics for Neurological Diseases

UMMS Affiliation

RNA Therapeutics Institute; Department of Neurology

Publication Date

2019-04-29

Document Type

Editorial

Disciplines

Biochemistry, Biophysics, and Structural Biology | Genetics and Genomics | Nervous System Diseases | Nucleic Acids, Nucleotides, and Nucleosides | Therapeutics

Abstract

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 [3] compounds reaching approval.

DOI of Published Version

10.1007/s13311-019-00736-1

Source

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

Journal/Book/Conference Title

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics

Related Resources

Link to Article in PubMed

PubMed ID

31037650

Share

COinS