RNA interference as an anticancer therapy: a patent perspective

被引:22
作者
Dykxhoorn, Derek M. [1 ,2 ]
机构
[1] Univ Miami, Miller Sch Med, Miami Inst Human Genom, John T Macdonald Fdn,Dept Human Genet, Miami, FL 33136 USA
[2] Univ Miami, Miller Sch Med, Miami Inst Human Genom, John T Macdonald Fdn,Dept Microbiol & Immunol, Miami, FL 33136 USA
关键词
cancer; drug discovery; generic therapy; RNA interference; RECEPTOR TYROSINE KINASES; IN-VIVO DELIVERY; GENE-THERAPY; SIRNA DELIVERY; OCULAR NEOVASCULARIZATION; MULTIDRUG-RESISTANCE; INDUCED SUPPRESSION; MICRORNA GENES; STRANDED-RNA; CELL-GROWTH;
D O I
10.1517/13543770902838008
中图分类号
R914 [药物化学];
学科分类号
100701 ;
摘要
Background: The development of RNA interference (RNAi)-based gene silencing approaches has revolutionized biomedical research. These technologies have been applied for functional genomic studies in a variety of areas, including cancer research, by facilitating a better understanding of the mechanisms that underlie tumorigenicity and the identification of novel factors that either promote or inhibit oncogenic transformation. Objective: These approaches have laid the groundwork for the development of a novel class of genetic therapies. Preclinical results have exposed both the unique therapeutic opportunities and challenges that are encountered in adapting these technologies for clinical applications. These themes are reflected in the patent literature that has mirrored the rise in complexity and sophistication of RNAi-based therapeutic approaches. This review focuses on the identification of potential anticancer therapeutic targets and the development of clinically relevant delivery approaches. Conclusions: Thus far, the patent landscape in the RNAi field has been dominated by a handful of key patents that describe the original identification and characterization of inhibitory double-stranded RNA molecules. Only time will tell how these original patents will hold up in the face of the development of new approaches and reagents as RNAi-based therapeutics approach transition from the bench to the clinic.
引用
收藏
页码:475 / 491
页数:17
相关论文
共 197 条
[21]   Loss of the cylindromatosis tumour suppressor inhibits apoptosis by activating NF-κB [J].
Brummelkamp, TR ;
Nijman, SMB ;
Dirac, AMG ;
Bernards, R .
NATURE, 2003, 424 (6950) :797-801
[22]   Human microRNA genes are frequently located at fragile sites and genomic regions involved in cancers [J].
Calin, GA ;
Sevignani, C ;
Dan Dumitru, C ;
Hyslop, T ;
Noch, E ;
Yendamuri, S ;
Shimizu, M ;
Rattan, S ;
Bullrich, F ;
Negrini, M ;
Croce, CM .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2004, 101 (09) :2999-3004
[23]   Potential applications for RNAi to probe pathogenesis and develop new treatments for ocular disorders [J].
Campochiaro, PA .
GENE THERAPY, 2006, 13 (06) :559-562
[24]   Small interfering RNA-induced suppression of ERM enhances sensitivity of human cancer cells to cisplatin [J].
Chang, IY ;
Kim, MH ;
Kim, HB ;
Lee, DY ;
Kim, SH ;
Kim, HY ;
You, HJ .
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, 2005, 327 (01) :225-233
[25]   Stable expression of small interfering RNA sensitizes TEL-PDGFβR to inhibition with imatinib or rapamycin [J].
Chen, J ;
Wall, NR ;
Kocher, K ;
Duclos, N ;
Fabbro, D ;
Neuberg, D ;
Griffin, JD ;
Shi, Y ;
Gilliland, DG .
JOURNAL OF CLINICAL INVESTIGATION, 2004, 113 (12) :1784-1791
[26]   TRBP recruits the Dicer complex to Ago2 for microRNA processing and gene silencing [J].
Chendrimada, TP ;
Gregory, RI ;
Kumaraswamy, E ;
Norman, J ;
Cooch, N ;
Nishikura, K ;
Shiekhattar, R .
NATURE, 2005, 436 (7051) :740-744
[27]   Novel cationic cardiolipin analogue-based liposome for efficient DNA and small interfering RNA delivery in vitro and in vivo [J].
Chien, PY ;
Wang, JK ;
Carbonaro, D ;
Lei, S ;
Miller, B ;
Sheikh, S ;
Ali, SM ;
Ahmad, MU ;
Ahmad, I .
CANCER GENE THERAPY, 2005, 12 (03) :321-328
[28]   miR-15 and miR-16 induce apoptosis by targeting BCL2 [J].
Cimmino, A ;
Calin, GA ;
Fabbri, M ;
Iorio, MV ;
Ferracin, M ;
Shimizu, M ;
Wojcik, SE ;
Aqeilan, RI ;
Zupo, S ;
Dono, M ;
Rassenti, L ;
Alder, H ;
Volinia, S ;
Liu, CG ;
Kipps, TJ ;
Negrini, M ;
Croce, CM .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2005, 102 (39) :13944-13949
[29]   Molecular origins of cancer: Oncogenes and cancer [J].
Croce, Carlo M. .
NEW ENGLAND JOURNAL OF MEDICINE, 2008, 358 (05) :502-511
[30]   An endogenous small interfering RNA pathway in Drosophila [J].
Czech, Benjamin ;
Malone, Colin D. ;
Zhou, Rui ;
Stark, Alexander ;
Schlingeheyde, Catherine ;
Dus, Monica ;
Perrimon, Norbert ;
Kellis, Manolis ;
Wohlschlegel, James A. ;
Sachidanandam, Ravi ;
Hannon, Gregory J. ;
Brennecke, Julius .
NATURE, 2008, 453 (7196) :798-U7