The silent treatment: SiRNAs as small molecule drugs

D. M. Dykxhoorn; D. Palliser; J. Lieberman

doi:10.1038/sj.gt.3302703

The silent treatment: SiRNAs as small molecule drugs

D. M. Dykxhoorn, D. Palliser, J. Lieberman

Research output: Contribution to journal › Review article › peer-review

309 Scopus citations

Abstract

As soon as RNA interference (RNAi) was found to work in mammalian cells, research quickly focused on harnessing this powerful endogenous and specific mechanism of gene silencing for human therapy. RNAi uses small RNAs, less than 30 nucleotides in length, to suppress expression of genes with complementary sequences. Two strategies can introduce small RNAs into the cytoplasm of cells, where they are active - a drug approach where double-stranded RNAs are administered in complexes designed for intracellular delivery and a gene therapy approach to express precursor RNAs from viral vectors. Phase I clinical studies have already begun to test the therapeutic potential of small RNA drugs that silence disease-related genes by RNAi. This review will discuss progress in developing and testing small RNAi-based drugs and potential obstacles.

Original language	English (US)
Pages (from-to)	541-552
Number of pages	12
Journal	Gene Therapy
Volume	13
Issue number	6
DOIs	https://doi.org/10.1038/sj.gt.3302703
State	Published - Mar 2006
Externally published	Yes

Keywords

Drug development
RNA interference
Small interfering RNA
Therapy

ASJC Scopus subject areas

Molecular Medicine
Molecular Biology
Genetics

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

Access to Document

10.1038/sj.gt.3302703

Cite this

@article{24cc6b4efc924923bec45345e4d906e8,

title = "The silent treatment: SiRNAs as small molecule drugs",

abstract = "As soon as RNA interference (RNAi) was found to work in mammalian cells, research quickly focused on harnessing this powerful endogenous and specific mechanism of gene silencing for human therapy. RNAi uses small RNAs, less than 30 nucleotides in length, to suppress expression of genes with complementary sequences. Two strategies can introduce small RNAs into the cytoplasm of cells, where they are active - a drug approach where double-stranded RNAs are administered in complexes designed for intracellular delivery and a gene therapy approach to express precursor RNAs from viral vectors. Phase I clinical studies have already begun to test the therapeutic potential of small RNA drugs that silence disease-related genes by RNAi. This review will discuss progress in developing and testing small RNAi-based drugs and potential obstacles.",

keywords = "Drug development, RNA interference, Small interfering RNA, Therapy",

author = "Dykxhoorn, {D. M.} and D. Palliser and J. Lieberman",

note = "Funding Information: This work was supported by NIH grants AI058695 and AI056900. We thank members of the laboratory and our collaborators for many useful suggestions.",

year = "2006",

month = mar,

doi = "10.1038/sj.gt.3302703",

language = "English (US)",

volume = "13",

pages = "541--552",

journal = "Gene Therapy",

issn = "0969-7128",

publisher = "Nature Publishing Group",

number = "6",

}

TY - JOUR

T1 - The silent treatment

T2 - SiRNAs as small molecule drugs

AU - Dykxhoorn, D. M.

AU - Palliser, D.

AU - Lieberman, J.

N1 - Funding Information: This work was supported by NIH grants AI058695 and AI056900. We thank members of the laboratory and our collaborators for many useful suggestions.

PY - 2006/3

Y1 - 2006/3

N2 - As soon as RNA interference (RNAi) was found to work in mammalian cells, research quickly focused on harnessing this powerful endogenous and specific mechanism of gene silencing for human therapy. RNAi uses small RNAs, less than 30 nucleotides in length, to suppress expression of genes with complementary sequences. Two strategies can introduce small RNAs into the cytoplasm of cells, where they are active - a drug approach where double-stranded RNAs are administered in complexes designed for intracellular delivery and a gene therapy approach to express precursor RNAs from viral vectors. Phase I clinical studies have already begun to test the therapeutic potential of small RNA drugs that silence disease-related genes by RNAi. This review will discuss progress in developing and testing small RNAi-based drugs and potential obstacles.

AB - As soon as RNA interference (RNAi) was found to work in mammalian cells, research quickly focused on harnessing this powerful endogenous and specific mechanism of gene silencing for human therapy. RNAi uses small RNAs, less than 30 nucleotides in length, to suppress expression of genes with complementary sequences. Two strategies can introduce small RNAs into the cytoplasm of cells, where they are active - a drug approach where double-stranded RNAs are administered in complexes designed for intracellular delivery and a gene therapy approach to express precursor RNAs from viral vectors. Phase I clinical studies have already begun to test the therapeutic potential of small RNA drugs that silence disease-related genes by RNAi. This review will discuss progress in developing and testing small RNAi-based drugs and potential obstacles.

KW - Drug development

KW - RNA interference

KW - Small interfering RNA

KW - Therapy

UR - http://www.scopus.com/inward/record.url?scp=33645148088&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=33645148088&partnerID=8YFLogxK

U2 - 10.1038/sj.gt.3302703

DO - 10.1038/sj.gt.3302703

M3 - Review article

C2 - 16397510

AN - SCOPUS:33645148088

SN - 0969-7128

VL - 13

SP - 541

EP - 552

JO - Gene Therapy

JF - Gene Therapy

IS - 6

ER -

The silent treatment: SiRNAs as small molecule drugs

Abstract

Keywords

ASJC Scopus subject areas

UN SDGs

Access to Document

Other files and links

Fingerprint

Cite this