Web Focus
The development of RNAi as a therapeutic strategy
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- Nature
- Nature Medicine
- Nature Reviews Genetics
- Nature Reviews Drug Discovery
- Nature Chemical Biology
- Nature Reviews Molecular Chemical Biology
- Nature Clinical Practice Neurology
- Laboratory Investigation
- British Journal of Cancer
- British Journal of Pharmacology
- Clinical Pharmacology & Therapeutics
- Cancer Gene Therapy
- Gene Therapy
- Journal of Investigative Dermatology
- Over 30 pharmaceutical and biotechnology companies have declared an interest in or have an active drug development program already underway in RNAi-based therapeutics to silence disease associated genes. This web focus collects together the latest research covering the development of RNAi based tools for drug target and gene function analysis as well as work describing the development of the technology for therapeutic applications.
Top of page
: 2008
Efficient Oncogene Silencing and Metastasis Inhibition via Systemic Delivery of siRNA
Shyh-Dar Li, Sumio Chono and Leaf Huang
Molecular Therapy 16: 942-946; doi:10.1038/mt.2008.51
AAV Vector-mediated RNAi of Mutant Huntingtin Expression Is Neuroprotective in a Novel Genetic Rat Model of Huntington's Disease
Nicholas R Franich, Helen L Fitzsimons, Dahna M Fong, Matthias Klugmann, Matthew J During and Deborah Young
Molecular Therapy 16: 947-956; doi:10.1038/mt.2008.50
Lentiviral Vector Design for Multiple shRNA Expression and Durable HIV-1 Inhibition
Kazutaka Nishina, Toshinori Unno, Yoshitaka Uno, Takayuki Kubodera, Tadashi Kanouchi, Hidehiro Mizusawa and Takanori Yokota
Molecular Therapy 16: 557-564; doi:10.1038/sj.mt.6300382
Titers of HIV-based Vectors Encoding shRNAs are Reduced by a Dicer-dependent Mechanism
Kazutaka Nishina, Toshinori Unno, Yoshitaka Uno, Takayuki Kubodera, Tadashi Kanouchi, Hidehiro Mizusawa and Takanori Yokota
Molecular Therapy 16: 378-386; doi:10.1038/sj.mt.6300370
: 2007
Top of pageSpecific inhibition of HBV replication in vitro and in vivo with expressed long hairpin RNA
Weinberg, M.S., Ely, A., Barichievy, S., Crowther, C., Mufamadi, S., Carmona, S., Arbuthnot, P.
Molecular Therapy 15: 534-541; doi:10.1038/sj.mt.6300077
Combinatorial RNAi: A winning strategy for the race against evolving targets?
Grimm, D., Kay, M.A.
Molecular Therapy 15: 878-888; doi:10.1038/sj.mt.6300116
: 2006
Top of pageProgress towards in vivo use of siRNAs
Behlke, M.A.
Molecular Therapy 13: 644-670; doi:10.1016/j.ymthe.2006.01.001
Design of noninflammatory synthetic siRNA mediating potent gene silencing in vivo
Judge, A.D., Bola, G., Lee, A.C.H., MacLachlan, I.
Molecular Therapy 13: 494-505; 10.1016/j.ymthe.2005.11.002
Silencing of HIV-1 with RNA Interference: a Multiple shRNA Approach
ter Brake, O., Konstantinova, P., Ceylan, M., Berkhout, B.
Molecular Therapy 14: 883-892; doi:10.1016/j.ymthe.2006.07.007
RNA Interference in Vitro and in Vivo Using a Novel Chitosan/siRNA Nanoparticle System
Howard, K.A., Rahbek, U.L., Liu, X., Damgaard,C.K., Glud, S.Z., Andersen,M.Ø, Hovgaard, M.B., Schmitz, A., Nyengaard, J.R., Besenbacher,F. and Kjems, J.
Molecular Therapy 14: 476-484; doi:10.1016/j.ymthe.2006.04.010
: Nature
Top of pageSequence- and target-independent angiogenesis suppression by siRNA via TLR3
Mark E. Kleinman, Kiyoshi Yamada, Atsunobu Takeda, Vasu Chandrasekaran, Miho Nozaki, Judit Z. Baffi, Romulo J. C. Albuquerque, Satoshi Yamasaki, Masahiro Itaya, Yuzhen Pan, Binoy Appukuttan, Daniel Gibbs, Zhenglin Yang, Katalin Karikó, Balamurali K. Ambati, Traci A. Wilgus, Luisa A. DiPietro, Eiji Sakurai, Kang Zhang, Justine R. Smith, Ethan W. Taylor & Jayakrishna Ambati.
Nature 452: 591-597; doi:10.1038/nature06765
Transvascular delivery of small interfering RNA to the central nervous system
John Witte
Nature 448: 39-43; doi:10.1038/nature05901
: Nature Medicine
Top of pageIn vivo imaging of siRNA delivery and silencing in tumors
Zdravka Medarova, Wellington Pham, Christian Farrar, Victoria Petkova, Anna Moore
Nature Medicine 13: 372 - 377; doi:10.1038/nm1486
: Nature Reviews Genetics
Top of pageStrategies for silencing human disease using RNA interference
Daniel H. Kim, John J. Rossi
Nature Reviews Genetics 8: 173-184; doi:10.1038/nrg2006
Genetic medicines: treatment strategies for hereditary disorders
Stuart J. Ellem and Gail P. Risbridger
Nature Reviews Genetics 7: 261-276; doi:10.1038/nrg1829
: Nature Reviews Drug Discovery
Top of pageInterfering with disease: a progress report on siRNA-based therapeutics
Antonin de Fougerolles, Hans-Peter Vornlocher, John Maraganore & Judy Lieberman
Nature Reviews Drug Discovery 6: 443-453; doi:10.1038/nrd2310
: Nature Chemical Biology
Top of pageRNAi therapeutics: a potential new class of pharmaceutical drugs
David Bumcrot, Muthiah Manoharan, Victor Koteliansky and Dinah W Y Sah
Nature Chemical Biology 2: 711-719; doi:10.1038/nchembio839
: Nature Reviews Molecular Cell Biology
Top of pageIlluminating the silence: understanding the structure and function of small RNAs
Tariq M. Rana
Nature Reviews Molecular Cell Biology 8: 23-36; doi:10.1038/nrm2085
: Nature Clinical Practice Neurology
Top of pageTechnology Insight: therapeutic RNA interference-how far from the neurology clinic?
Pedro Gonzalez-Alegre and Henry L Paulson
Nature Clinical Practice Neurology 3: 394-404; doi:10.1038/ncpneuro0551
: Laboratory Investigation
Top of pageIn vivo post-transcriptional gene silencing of a-1 antitrypsin by adeno-associated virus vectors expressing siRNA
Pedro E Cruz, Christian Mueller, Travis L Cossette, Alexandra Golant, Qiushi Tang, Stuart G Beattie, Mark Brantly, Martha Campbell-Thompson, Keith S Blomenkamp, Jeffrey H Teckman and Terence R Flotte
Laboratory Investigation 87: 893-902; doi:10.1038/labinvest.3700629
Everything you wanted to know about small RNA but were afraid to ask
Scott D Boyd
Laboratory Investigation 88; 569-578; doi: 10.1038/labinvest.2008.32
: British Journal of Cancer
Top of pageEnhancement of radiation response by inhibition of Aurora-A kinase using siRNA or a selective Aurora kinase inhibitor PHA680632 in p53-deficient cancer cells
Lecluse Y, Auperin A, Bourhis J, Deutsch E
British Journal of Cancer 97: 1664-1672; doi:10.1038/sj.bjc.6604083
RNA interference as a key to knockdown overexpressed cyclooxygenase-2 gene in tumour cells
Strillacci A, Griffoni C, Spisni E, Manara MC, Tomasi V
British Journal of Cancer 94: 1300-1310; doi:10.1038/sj.bjc.6603094
: British Journal of Pharmacology
Top of pageInvestigation of the role of TASK-2 channels in rat pulmonary arteries; pharmacological and functional studies following RNA interference procedures
Gonczi M, Szentandrassy N, Johnson IT, Heagerty AM, Weston AH
British Journal of Pharmacology 147: 496-505; doi:10.1038/sj.bjp.0706649
: Clinical Pharmacology & Therapeutics
Top of pageCommentary: 'Drug Discovery and Development in the Age of Molecular Medicine'
P Vallance and M Levick
Clinical Pharmacology & Therapeutics 82: 363-366; doi:10.1038/sj.clpt.610033
: Cancer Gene Therapy
Top of pagesiRNA-based approaches in cancer therapy
G R Devi
Cancer Gene Therapy 13: 819-829 ; doi:10.1038/sj.cgt.7700931
New highly potent and specific E6 and E7 siRNAs for treatment of HPV16 positive cervical cancer
K Yamato, T Yamada, M Kizaki, K Ui-Tei, Y Natori, M Fujino, T Nishihara, Y Ikeda, Y Nasu, K Saigo, M Yoshinouchi
Cancer Gene Therapy 15: 140-153; doi:10.1038/sj.cgt.7701118
: Gene Therapy
Top of pageAtelocollagen-mediated local and systemic applications of myostatin-targeting siRNA increase skeletal muscle mass
Kinouchi N, Ohsawa Y, Ishimaru N, Ohuchi H, Sunada Y, Hayashi Y, Tanimoto Y, Moriyama K, Noji S
Gene Therapy advance online publication; doi:10.1038/gt.2008.24
Increased virus replication in mammalian cells by blocking intracellular innate defense responses
de Vries W, Haasnoot J, van der Velden J, van Montfort T, Zorgdrager F, Paxton W, Cornelissen M, van Kuppeveld F, de Haan P, Berkhout B.
Gene Therapy 15: 545-52; doi:10.1038/gt.2008.12
: Journal of Investigative Dermatology
Top of pageDevelopment of Therapeutic siRNAs for Pachyonychia Congenita
Frances J D Smith, Robyn P Hickerson, Jane M Sayers, Robert E Reeves, Christopher H Contag, Devin Leake, Roger L Kaspar and W H Irwin McLean
Journal of Investigative Dermatology 128: 50-58; doi:10.1038/sj.jid.5701040
Delivery and Inhibition of Reporter Genes by Small Interfering RNAs in a Mouse Skin Model
Qian Wang, Heini Ilves, Pauline Chu, Christopher H Contag, Devin Leake, Brian H Johnston and Roger L Kaspar
Journal of Investigative Dermatology 127: 2577-2584; doi:10.1038/sj.jid.5700891
