Nucleic Acid Therapeutics for Neurological Diseases
Watts, Jonathan K ; Brown, Robert H. Jr. ; Khvorova, Anastasia
Watts, Jonathan K
Brown, Robert H. Jr.
Khvorova, Anastasia
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UMass Chan Affiliations
Document Type
Editorial
Publication Date
2019-04-29
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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
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DOI
10.1007/s13311-019-00736-1
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PubMed ID
31037650