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. 2014 Jun 11;6(240):240ps7.
doi: 10.1126/scitranslmed.3008362.

RNAi therapies: drugging the undruggable

Sherry Y Wu  1 , Gabriel Lopez-Berestein  2 , George A Calin  2 , Anil K Sood  3
Affiliations

RNAi therapies: drugging the undruggable

Sherry Y Wu et al. Sci Transl Med. .

Abstract

RNA interference (RNAi) therapy is a rapidly emerging platform for personalized cancer treatment. Recent advances in small interfering RNA delivery and target selection provide unprecedented opportunities for clinical translation. Here, we discuss these advances and present strategies for making RNAi-based therapy a viable part of cancer management.

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Figures

Fig. 1
Fig. 1
Overcoming obstacles: critical aspects to consider when designing effective RNAi-based cancer therapeutics.
See this image and copyright information in PMC

References

    1. Tabernero SG, LoRusso PM, Cervantes A, Schwartz GK, Weiss GJ, Paz-Ares L, Cho DC, Infante JR, Alsina M, Gounder MM, Falzone R, Harrop J, White AC, Toudjarska I, Bumcrot D, Meyers RE, Hinkle G, Svrzikapa N, Hutabarat RM, Clausen VA, Cehelsky J, Nochur SV, Gamba-Vitalo C, Vaishnaw AK, Sah DW, Gollob JA, Burris HA., 3rd First-in-humans trial of an RNA interference therapeutic targeting VEGF and KSP in cancer patients with liver involvement. Cancer Discov. 2013;3:406–417. - PubMed
    1. Golan T, Hubert A, Shemi A, Segal A, Dancour A, Khvalevsky EZ. A phase I trial of a local delivery of siRNA against k-ras in combination with chemotherapy for locally advanced pancreatic adenocarcinoma. Paper presented at the 49th Annual Meeting of the American Society of Clinical Oncology; Chicago, IL. 31 May to 4 June, 2013.
    1. Davis ME, Zuckerman JE, Choi CH, Seligson D, Tolcher A, Alabi CA, Yen Y, Heidel JD, Ribas A. Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles. Nature. 2010;646:1067–1070. - PMC - PubMed
    1. Coelho T, Adams D, Silva A, Lozeron P, Hawkins PN, Mant T, Perez J, Chiesa J, Warrington S, Tranter E, Munisamy M, Falzone R, Harrop J, Cehelsky J, Bettencourt BR, Geissler M, Butler JS, Sehgal A, Meyers RE, Chen Q, Borland T, Hutabarat RM, Clausen VA, Alvarez R, Fitzgerald K, Gamba-Vital C, Nochur SV, Vaishnaw AK, Sah DW, Gollob JA, Suhr OB. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med. 2013;369:819–829. - PubMed
    1. Strumberg D, Schultheis B, Traugott U, Vank C, Santel A, Keil O, Kaufmann J, Drevs J. Phase I clinical development of Atu027, a siRNA formulation targeting PKN3 in patients with advanced solid tumors. Int J Clin Pharmacol Ther. 2012;50:76–78. - PubMed

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