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PMID: 18060021 Published · ppublish English Journal Article Research Support, N.I.H., Extramural Review

Therapeutic application of RNAi: is mRNA targeting finally ready for prime time?

The Journal of clinical investigation ·Vol. 117 ·No. 12 ·2007-12-00 ·Pages 3633-41

Grimm D, Kay MA

Abstract

With unprecedented speed, RNA interference (RNAi) has advanced from its basic discovery in lower organisms to becoming a powerful genetic tool and perhaps our single most promising biotherapeutic for a wide array of diseases. Numerous studies document RNAi efficacy in laboratory animals, and the first clinical trials are underway and thus far suggest that RNAi is safe to use in humans. Yet substantial hurdles have also surfaced and must be surmounted before therapeutic RNAi applications can become a standard therapy. Here we review the most critical roadblocks and concerns for clinical RNAi transition, delivery, and safety. We highlight emerging solutions and concurrently discuss novel therapeutic RNAi-based concepts. The current rapid advances create realistic optimism that the establishment of RNAi as a new and potent clinical modality in humans is near.

MeSH Terms
Animals Clinical Trials as Topic Drug Delivery Systems Drug-Related Side Effects and Adverse Reactions Humans RNA Interference/drug effects RNA, Messenger/antagonists & inhibitors RNA, Small Interfering/adverse effects,chemistry,therapeutic use
Chemicals
RNA, Messenger RNA, Small Interfering
Authors & Affiliations
2 authors, click to expand affiliations / ORCID
Grimm Dirk
Department of Pediatrics, Stanford University School of Medicine, Stanford, California 94305, USA.
Kay Mark A
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Article Info
Journal
The Journal of clinical investigation
Abbr.
J Clin Invest
ISSN
0021-9738
DOI
10.1172/JCI34129
Published
2007-12-00
Pages
3633-41
Language
English
Region
United States
NLM ID
7802877
PMCID
PMC2096424
Subset
IM
Grants
NIAID NIH HHS · R01 AI071068 · United States
NIDDK NIH HHS · R01 DK078424 · United States
NIDDK NIH HHS · DK078424 · United States
NIAID NIH HHS · AI071068 · United States
External Links
PubMed source DOI: 10.1172/JCI34129 PMC Full Text View journal details
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