Subscribe to Journal
Get full journal access for 1 year
only $102.00 per issue
All prices are NET prices.
VAT will be added later in the checkout.
Rent or Buy article
Get time limited or full article access on ReadCube.
All prices are NET prices.
Coelho T, Adams D, Silva A, Lozeron P, Hawkins PN, Mant T et al. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med 2013; 369: 819–829.
Fitzgerald K, Kallend D, Simon A . A highly durable RNAi therapeutic inhibitor of PCSK9. N Engl J Med 2017; 376: e38.
Rungta RL, Choi HB, Lin PJ, Ko RW, Ashby D, Nair J et al. Lipid nanoparticle delivery of siRNA to silence neuronal gene expression in the brain. Mol Ther Nucleic Acids 2013; 2: e136.
Mitelman F, Johansson B, Mertens F . The impact of translocations and gene fusions on cancer causation. Nat Rev Cancer 2007; 7: 233–245.
Hunger SP, Mullighan CG . Acute lymphoblastic leukemia in children. N Engl J Med 2015; 373: 1541–1552.
Howard KA, Rahbek UL, Liu X, Damgaard CK, Glud SZ, Andersen MO et al. RNA interference in vitro and in vivo using a novel chitosan/siRNA nanoparticle system. Mol Ther 2006; 14: 476–484.
Fleischmann KK, Pagel P, Schmid I, Roscher AA . RNAi-mediated silencing of MLL-AF9 reveals leukemia-associated downstream targets and processes. Mol Cancer 2014; 13: 27.
Takenaka S, Naka N, Araki N, Hashimoto N, Ueda T, Yoshioka K et al. Downregulation of SS18-SSX1 expression in synovial sarcoma by small interfering RNA enhances the focal adhesion pathway and inhibits anchorage-independent growth in vitro and tumor growth in vivo. Int J Oncol 2010; 36: 823–831.
Urbinati G, Ali HM, Rousseau Q, Chapuis H, Desmaele D, Couvreur P et al. Antineoplastic effects of siRNA against TMPRSS2-ERG junction oncogene in prostate cancer. PLoS ONE 2015; 10: e0125277.
Koldehoff M, Steckel NK, Beelen DW, Elmaagacli AH . Therapeutic application of small interfering RNA directed against bcr-abl transcripts to a patient with imatinib-resistant chronic myeloid leukaemia. Clin Exp Med 2007; 7: 47–55.
Heim SMF . Cancer Cytogenetics: Chromosomal and Molecular Genetic Aberrations of Tumor Cells,4th edn.Wiley-Blackwell, 2015.
Felice MS, Gallego MS, Alonso CN, Alfaro EM, Guitter MR, Bernasconi AR et al. Prognostic impact of t(1;19)/ TCF3-PBX1 in childhood acute lymphoblastic leukemia in the context of Berlin-Frankfurt-Munster-based protocols. Leuk Lymphoma 2011; 52: 1215–1221.
Fischer U, Forster M, Rinaldi A, Risch T, Sungalee S, Warnatz HJ et al. Genomics and drug profiling of fatal TCF3-HLF-positive acute lymphoblastic leukemia identifies recurrent mutation patterns and therapeutic options. Nat Genet 2015; 47: 1020–1029.
Weinstein S, Toker IA, Emmanuel R, Ramishetti S, Hazan-Halevy I, Rosenblum D et al. Harnessing RNAi-based nanomedicines for therapeutic gene silencing in B-cell malignancies. Proc Natl Acad Sci USA 2016; 113: E16–E22.
Jin SE, Jin HE, Hong SS . Targeted delivery system of nanobiomaterials in anticancer therapy: from cells to clinics. Biomed Res Int 2014; 2014: 814208.
We thank Silke Glowotz, Martin Wichmann, Anitha Thomas and Colin Walsh for technical support. We express sincere thanks to the patients for their participation in the study. We thank Prof. Dr Alf Lamprecht and Dr Manusmriti Singh for providing us nanoparticles during the initial experiments. We thank the staff of the Central Animal Facility and Matthias Ballmaier from the Cell Sorting Core Facility (supported in part by the Braukmann-Wittenberg-Herz-Stiftung and the Deutsche Forschungsgemeinschaft) of Hannover Medical School. This study was supported by the Rudolf-Bartling Stiftung, an ERC grant under the European Union’s Horizon 2020 research and innovation programme (No. 638035), by grants 110284, 110287, 110292 and 111267 from Deutsche Krebshilfe; grant DJCLS R13/14 from the Deutsche José Carreras Leukämie-Stiftung e.V; the German Federal Ministry of Education and Research grant 01EO0802 (IFB-Tx); DFG grant HE 5240/5-1, HE 5240/5-2 and HE 5240/6-1; grants from Dieter-Schlag Stiftung and a Terry Fox Foundation Program Project Award to RKH.
Euan Ramsay is an employee of Precision Nanosystems. Pieter Cullis is founder of Precision Nanosystems. The other authors have no conflicts of interest.
Supplementary Information accompanies this paper on the Leukemia website
About this article
Cite this article
Jyotsana, N., Sharma, A., Chaturvedi, A. et al. RNA interference efficiently targets human leukemia driven by a fusion oncogene in vivo. Leukemia 32, 224–226 (2018) doi:10.1038/leu.2017.269
Nature Reviews Drug Discovery (2019)
Expert Opinion on Therapeutic Targets (2019)
Annals of Hematology (2019)
Exploiting differential RNA splicing patterns: a potential new group of therapeutic targets in cancer
Expert Opinion on Therapeutic Targets (2018)
Impedimetric gene assay for BCR/ABL transcripts in plasmids of patients with chronic myeloid leukemia
Microchimica Acta (2018)