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Knocking down disease: a progress report on siRNA therapeutics.

Wittrup, Anders LU and Lieberman, Judy (2015) In Nature Reviews. Genetics 16(9). p.543-552
Abstract
Small interfering RNAs (siRNAs), which downregulate gene expression guided by sequence complementarity, can be used therapeutically to block the synthesis of disease-causing proteins. The main obstacle to siRNA drugs - their delivery into the target cell cytosol - has been overcome to allow suppression of liver gene expression. Here, we review the results of recent clinical trials of siRNA therapeutics, which show efficient and durable gene knockdown in the liver, with signs of promising clinical outcomes and little toxicity. We also discuss the barriers to more widespread applications that target tissues besides the liver and the most promising avenues to overcome them.
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Nature Reviews. Genetics
volume
16
issue
9
pages
543 - 552
publisher
Nature Publishing Group
external identifiers
  • wos:000359954900009
  • pmid:26281785
  • scopus:84934307590
ISSN
1471-0056
DOI
10.1038/nrg3978
language
English
LU publication?
yes
id
234bc14d-6412-48d9-a9bf-b0810a5ddf84 (old id 7840765)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/26281785?dopt=Abstract
date added to LUP
2015-09-06 14:39:00
date last changed
2017-11-05 03:24:59
@article{234bc14d-6412-48d9-a9bf-b0810a5ddf84,
  abstract     = {Small interfering RNAs (siRNAs), which downregulate gene expression guided by sequence complementarity, can be used therapeutically to block the synthesis of disease-causing proteins. The main obstacle to siRNA drugs - their delivery into the target cell cytosol - has been overcome to allow suppression of liver gene expression. Here, we review the results of recent clinical trials of siRNA therapeutics, which show efficient and durable gene knockdown in the liver, with signs of promising clinical outcomes and little toxicity. We also discuss the barriers to more widespread applications that target tissues besides the liver and the most promising avenues to overcome them.},
  author       = {Wittrup, Anders and Lieberman, Judy},
  issn         = {1471-0056},
  language     = {eng},
  number       = {9},
  pages        = {543--552},
  publisher    = {Nature Publishing Group},
  series       = {Nature Reviews. Genetics},
  title        = {Knocking down disease: a progress report on siRNA therapeutics.},
  url          = {http://dx.doi.org/10.1038/nrg3978},
  volume       = {16},
  year         = {2015},
}