Naked antisense double-stranded DNA oligonucleotide efficiently suppresses BCR-ABL positive leukemic cells | springermedizin.de

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Investigational New Drugs

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24.10.2019 | PRECLINICAL STUDIES

Naked antisense double-stranded DNA oligonucleotide efficiently suppresses BCR-ABL positive leukemic cells

verfasst von: Toshimi Hoshiko, Yasushi Kubota, Takuya Akisawa, Tatsuro Watanabe, Kazuaki Tanigawara, Junichi Yano, Shinya Kimura

Erschienen in: Investigational New Drugs | Ausgabe 4/2020

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Summary

Oligonucleotide-based gene silencing, using molecules such as antisense oligonucleotides (ASOs), small interfering RNA, and aptamers, is widely studied. Another approach uses DNA/RNA heteroduplex oligonucleotides (HDOs). Here, we developed an antisense double-stranded DNA oligonucleotide (ADO) by modification of the complementary RNA in an HDO to generate DNA for increasing resistance to nucleases. Naked BCR-ABL-targeting ADO was significantly more potent than siRNA at reducing BCR-ABL chimeric mRNA expression in chronic myeloid leukemia (CML) cell lines. Further, naked BCR-ABL-targeting ADO suppressed BCR-ABL protein levels in a dose-dependent manner, inhibited CML cell proliferation, and augmented the inhibitory effects of imatinib mesylate. In conclusion, ADO technology is an attractive method for therapeutic application.

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1.

Kimura S, Ando T, Kojima K (2014) Ever-advancing chronic myeloid leukemia treatment. Int J Clin Oncol 19(1):3–9. https://doi.org/10.1007/s10147-013-0641-7 CrossRefPubMed

2.

Baccarani M, Castagnetti F, Gugliotta G, Rosti G, Soverini S, Albeer A, Pfirrmann M (2019) The proportion of different BCR-ABL1 transcript types in chronic myeloid leukemia. An international overview. Leukemia 33(5):1173–1183. https://doi.org/10.1038/s41375-018-0341-4 CrossRefPubMed

3.

Druker BJ, Tamura S, Buchdunger E, Ohno S, Segal GM, Fanning S, Zimmermann J, Lydon NB (1996) Effects of a selective inhibitor of the Abl tyrosine kinase on the growth of Bcr-Abl positive cells. Nat Med 2(5):561–566CrossRef

4.

O'Brien SG, Guilhot F, Larson RA, Gathmann I, Baccarani M, Cervantes F, Cornelissen JJ, Fischer T, Hochhaus A, Hughes T, Lechner K, Nielsen JL, Rousselot P, Reiffers J, Saglio G, Shepherd J, Simonsson B, Gratwohl A, Goldman JM, Kantarjian H, Taylor K, Verhoef G, Bolton AE, Capdeville R, Druker BJ, I. Investigators (2003) Imatinib compared with interferon and low-dose cytarabine for newly diagnosed chronic-phase chronic myeloid leukemia, N Engl J Med 348(11):994-1004. https://doi.org/10.1056/NEJMoa022457

5.

Geary RS, Baker BF, Crooke ST (2015) Clinical and preclinical pharmacokinetics and pharmacodynamics of mipomersen (kynamro((R))): a second-generation antisense oligonucleotide inhibitor of apolipoprotein B. Clin Pharmacokinet 54(2):133–146. https://doi.org/10.1007/s40262-014-0224-4 CrossRefPubMedPubMedCentral

6.

Mendell JR, Rodino-Klapac LR, Sahenk Z, Roush K, Bird L, Lowes LP, Alfano L, Gomez AM, Lewis S, Kota J, Malik V, Shontz K, Walker CM, Flanigan KM, Corridore M, Kean JR, Allen HD, Shilling C, Melia KR, Sazani P, Saoud JB, Kaye EM, Eteplirsen Study Group (2013) Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann Neurol 74(5):637–647. https://doi.org/10.1002/ana.23982 CrossRefPubMed

7.

Chiriboga CA, Swoboda KJ, Darras BT, Iannaccone ST, Montes J, De Vivo DC, Norris DA, Bennett CF, Bishop KM (2016) Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy. Neurology 86(10):890–897. https://doi.org/10.1212/WNL.0000000000002445 CrossRefPubMedPubMedCentral

8.

Benson MD, Waddington-Cruz M, Berk JL, Polydefkis M, Dyck PJ, Wang AK, Planté-Bordeneuve V, Barroso FA, Merlini G, Obici L, Scheinberg M, Brannagan TH 3rd, Litchy WJ, Whelan C, Drachman BM, Adams D, Heitner SB, Conceição I, Schmidt HH, Vita G, Campistol JM, Gamez J, Gorevic PD, Gane E, Shah AM, Solomon SD, Monia BP, Hughes SG, Kwoh TJ, BW ME, Jung SW, Baker BF, Ackermann EJ, Gertz MA, Coelho T (2018) Inotersen treatment for patients with hereditary Transthyretin amyloidosis. N Engl J Med 379(1):22–31. https://doi.org/10.1056/NEJMoa1716793 CrossRefPubMed

9.

Adams D, Gonzalez-Duarte A, O'Riordan WD, Yang CC, Ueda M, Kristen AV, Tournev I, Schmidt HH, Coelho T, Berk JL, Lin KP, Vita G, Attarian S, Planté-Bordeneuve V, Mezei MM, Campistol JM, Buades J, Brannagan TH 3rd, Kim BJ, Oh J, Parman Y, Sekijima Y, Hawkins PN, Solomon SD, Polydefkis M, Dyck PJ, Gandhi PJ, Goyal S, Chen J, Strahs AL, Nochur SV, Sweetser MT, Garg PP, Vaishnaw AK, Gollob JA, Suhr OB (2018) Patisiran, an RNAi therapeutic, for hereditary Transthyretin amyloidosis. N Engl J Med 379(1):11–21. https://doi.org/10.1056/NEJMoa1716153 CrossRef

10.

Nishina K, Piao W, Yoshida-Tanaka K, Sujino Y, Nishina T, Yamamoto T, Nitta K, Yoshioka K, Kuwahara H, Yasuhara H, Baba T, Ono F, Miyata K, Miyake K, Seth PP, Low A, Yoshida M, Bennett CF, Kataoka K, Mizusawa H, Obika S, Yokota T (2015) DNA/RNA heteroduplex oligonucleotide for highly efficient gene silencing. Nat Commun 6:7969. https://doi.org/10.1038/ncomms8969 CrossRefPubMedPubMedCentral

11.

Asami Y, Yoshioka K, Nishina K, Nagata T, Yokota T (2016) Drug delivery system of therapeutic oligonucleotides. Drug Discov Ther 10(5):256–262. https://doi.org/10.5582/ddt.2016.01065 CrossRefPubMed

12.

Ito T, Tanaka H, Kimura A (2007) Establishment and characterization of a novel imatinib-sensitive chronic myeloid leukemia cell line MYL, and an imatinib-resistant subline MYL-R showing overexpression of Lyn. Eur J Haematol 78(5):417–431. https://doi.org/10.1111/j.1600-0609.2007.00835.x CrossRefPubMed

13.

Withey JM, Marley SB, Kaeda J, Harvey AJ, Crompton MR, Gordon MY (2005) Targeting primary human leukaemia cells with RNA interference: Bcr-Abl targeting inhibits myeloid progenitor self-renewal in chronic myeloid leukaemia cells. Br J Haematol 129(3):377–380. https://doi.org/10.1111/j.1365-2141.2005.05468.x CrossRefPubMed

14.

Amabile M, Giannini B, Testoni N, Montefusco V, Rosti G, Zardini C, Terragna C, Buonamici S, Ottaviani E, Soverini S, Fiacchini M, Bassi S, de Vivo A, Trabacchi E, Saglio G, Pane F, Baccarani M, Tura S, Martinelli G (2001) Real-time quantification of different types of bcr-abl transcript in chronic myeloid leukemia. Haematologica 86(3):252–259PubMed

15.

Nishida Y, Maeda A, Kim MJ, Cao L, Kubota Y, Ishizawa J, AlRawi A, Kato Y, Iwama A, Fujisawa M, Matsue K, Weetall M, Dumble M, Andreeff M, Davis TW, Branstrom A, Kimura S, Kojima K (2017) The novel BMI-1 inhibitor PTC596 downregulates MCL-1 and induces p53-independent mitochondrial apoptosis in acute myeloid leukemia progenitor cells. Blood Cancer J 7(2):e527. https://doi.org/10.1038/bcj.2017.8 CrossRefPubMedPubMedCentral

16.

Yokoo M, Kubota Y, Motoyama K, Higashi T, Taniyoshi M, Tokumaru H, Nishiyama R, Tabe Y, Mochinaga S, Sato A, Sueoka-Aragane N, Sueoka E, Arima H, Irie T, Kimura S (2015) 2-Hydroxypropyl-beta-Cyclodextrin acts as a novel anticancer agent. PLoS One 10(11):e0141946. https://doi.org/10.1371/journal.pone.0141946 CrossRefPubMedPubMedCentral

17.

Chow KU, Ries J, Weidmann E, Pourebrahim F, Napieralski S, Stieler M, Boehrer S, Rummel MJ, Stein J, Hoelzer D, Mitrou PS (2000) Induction of apoptosis using 2′,2′ difluorodeoxycytidine (gemcitabine) in combination with antimetabolites or anthracyclines on malignant lymphatic and myeloid cells. Antagonism or synergism depends on incubation schedule and origin of neoplastic cells. Ann Hematol 79(9):485–492CrossRef

18.

Kimura S, Kuroda J, Segawa H, Sato K, Nogawa M, Yuasa T, Ottmann OG, Maekawa T (2004) Antiproliferative efficacy of the third-generation bisphosphonate, zoledronic acid, combined with other anticancer drugs in leukemic cell lines. Int J Hematol 79(1):37–43CrossRef

19.

Kawata E, Ashihara E, Nakagawa Y, Kiuchi T, Ogura M, Yao H, Sakai K, Tanaka R, Nagao R, Yokota A, Takeuchi M, Kimura S, Hirai H, Maekawa T (2010) A combination of a DNA-chimera siRNA against PLK-1 and zoledronic acid suppresses the growth of malignant mesothelioma cells in vitro. Cancer Lett 294(2):245–253. https://doi.org/10.1016/j.canlet.2010.02.008 CrossRefPubMed

20.

Scherr M, Battmer K, Winkler T, Heidenreich O, Ganser A, Eder M (2003) Specific inhibition of bcr-abl gene expression by small interfering RNA. Blood 101(4):1566–1569. https://doi.org/10.1182/blood-2002-06-1685 CrossRefPubMed

21.

Koldehoff M, Steckel NK, Beelen DW, Elmaagacli AH (2007) Therapeutic application of small interfering RNA directed against bcr-abl transcripts to a patient with imatinib-resistant chronic myeloid leukaemia. Clin Exp Med 7(2):47–55. https://doi.org/10.1007/s10238-007-0125-z CrossRefPubMed

22.

de Fabritiis P, Petti MC, Montefusco E, De Propris MS, Sala R, Bellucci R, Mancini M, Lisci A, Bonetto F, Geiser T, Calabretta B, Mandelli F (1998) BCR-ABL antisense oligodeoxynucleotide in vitro purging and autologous bone marrow transplantation for patients with chronic myelogenous leukemia in advanced phase. Blood 91(9):3156–3162CrossRef

Titel: Naked antisense double-stranded DNA oligonucleotide efficiently suppresses BCR-ABL positive leukemic cells
verfasst von: Toshimi Hoshiko
Yasushi Kubota
Takuya Akisawa
Tatsuro Watanabe
Kazuaki Tanigawara
Junichi Yano
Shinya Kimura
Publikationsdatum: 24.10.2019
Verlag: Springer US
Erschienen in: Investigational New Drugs / Ausgabe 4/2020
Print ISSN: 0167-6997
Elektronische ISSN: 1573-0646
DOI: https://doi.org/10.1007/s10637-019-00862-9

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