Publication

Nucleic Acid Therapeutics for Neurological Diseases

Watts, Jonathan K
Brown, Robert H. Jr.
Khvorova, Anastasia
Embargo Expiration Date
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.

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

Year of Medical School at Time of Visit
Sponsors
Dates of Travel
DOI
10.1007/s13311-019-00736-1
PubMed ID
31037650
Other Identifiers
Notes
Funding and Acknowledgements
Corresponding Author
Related Resources
Related Resources
Repository Citation
Rights
Distribution License