Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2015 Jun 15:14:714-46.
doi: 10.17179/excli2015-297. eCollection 2015.

Short-interference RNAs: becoming medicines

Tamara Martínez  1 , Ana Isabel Jiménez  1 , Covadonga Pañeda  1
Affiliations

Short-interference RNAs: becoming medicines

Tamara Martínez et al. EXCLI J. .

Abstract

RNA interference is a cellular mechanism by which small molecules of double stranded RNA modulate gene expression acting on the concentration and/or availability of a given messenger RNA. Almost 10 years after Fire and Mello received the Nobel Prize for the discovery of this mechanism in flat worms, RNA interference is on the edge of becoming a new class of therapeutics. With various phase III studies underway, the following years will determine whether RNAi-therapeutics can rise up to the challenge and become mainstream medicines. The present review gives a thorough overview of the current status of this technology focusing on the path to the clinic of this new class of compounds.

Keywords: RNAi; oligonucleotides; siRNA; therapeutics.

PubMed Disclaimer

Figures

Table 1
Table 1. Classification of toxicities of siRNA-based compounds and recommendations to assess eventual toxicities
Table 2
Table 2. Programs in clinical development. (A) Active, (C) Complete and (T) Terminated
Table 3
Table 3. Programs in preclinical development
See this image and copyright information in PMC

References

    1. Ahmed Z, Kalinski H, Berry M, Almasieh M, Ashush H, Slager N, et al. Ocular neuroprotection by siRNA targeting caspase-2. Cell Death Dis. 2011;2:e173. - PMC - PubMed
    1. Akinc A, Querbes W, De S, Qin J, Frank-Kamenetsky M, Jayaprakash KN, et al. Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms. Mol Ther. 2010;18:1357–1364. - PMC - PubMed
    1. Allerson CR, Sioufi N, Jarres R, Prakash TP, Naik N, Berdeja A, et al. Fully 2'-modified oligonucleotide duplexes with improved in vitro potency and stability compared to unmodified small interfering RNA. J Med Chem. 2005;48:901–904. - PubMed
    1. Amarzguioui M, Holen T, Babaie E, Prydz H. Tolerance for mutations and chemical modifications in a siRNA. Nucleic Acids Res. 2003;31:589–595. - PMC - PubMed
    1. Arthur MJ. Reversibility of liver fibrosis and cirrhosis following treatment for hepatitis C. Gastroenterology. 2002;122:1525–1528. - PubMed

LinkOut - more resources