메뉴 건너뛰기




Volumn 38, Issue 8, 2016, Pages 738-745

Staurosporine allows dystrophin expression by skipping of nonsense-encoding exon

Author keywords

Chemical; Dystrophin; Exon skipping; Nonsense mutation; Staurosporine

Indexed keywords

DYSTROPHIN; ENZASTAURIN; MESSENGER RNA; MIDOSTAURIN; PHOSPHOTRANSFERASE INHIBITOR; STAUROSPORINE; TG 003; UNCLASSIFIED DRUG; CLK DUAL-SPECIFICITY KINASES; DESMIN; PROTEIN SERINE THREONINE KINASE; PROTEIN TYROSINE KINASE; STOP CODON;

EID: 84961778327     PISSN: 03877604     EISSN: 18727131     Source Type: Journal    
DOI: 10.1016/j.braindev.2016.03.003     Document Type: Article
Times cited : (6)

References (31)
  • 1
    • 77950793623 scopus 로고    scopus 로고
    • RNA-targeted splice-correction therapy for neuromuscular disease
    • [1] Wood, M.J., Gait, M.J., Yin, H., RNA-targeted splice-correction therapy for neuromuscular disease. Brain 133 (2010), 957–972.
    • (2010) Brain , vol.133 , pp. 957-972
    • Wood, M.J.1    Gait, M.J.2    Yin, H.3
  • 3
    • 84945127144 scopus 로고    scopus 로고
    • Modulators of alternative splicing as novel therapeutics in cancer
    • [3] Oltean, S., Modulators of alternative splicing as novel therapeutics in cancer. World J Clin Oncol 6 (2015), 92–95.
    • (2015) World J Clin Oncol , vol.6 , pp. 92-95
    • Oltean, S.1
  • 5
    • 84907991204 scopus 로고    scopus 로고
    • Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study
    • [5] Voit, T., Topaloglu, H., Straub, V., Muntoni, F., Deconinck, N., Campion, G., et al. Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study. Lancet Neurol 13 (2014), 987–996.
    • (2014) Lancet Neurol , vol.13 , pp. 987-996
    • Voit, T.1    Topaloglu, H.2    Straub, V.3    Muntoni, F.4    Deconinck, N.5    Campion, G.6
  • 6
    • 84931072604 scopus 로고    scopus 로고
    • Contributions of Japanese patients to development of antisense therapy for DMD
    • [6] Matsuo, M., Takeshima, Y., Nishio, H., Contributions of Japanese patients to development of antisense therapy for DMD. Brain Dev 38 (2016), 4–9.
    • (2016) Brain Dev , vol.38 , pp. 4-9
    • Matsuo, M.1    Takeshima, Y.2    Nishio, H.3
  • 7
    • 0030152856 scopus 로고    scopus 로고
    • Duchenne/Becker muscular dystrophy: from molecular diagnosis to gene therapy
    • [7] Matsuo, M., Duchenne/Becker muscular dystrophy: from molecular diagnosis to gene therapy. Brain Dev 18 (1996), 167–172.
    • (1996) Brain Dev , vol.18 , pp. 167-172
    • Matsuo, M.1
  • 8
    • 69749110302 scopus 로고    scopus 로고
    • Less is more: therapeutic exon skipping for Duchenne muscular dystrophy
    • [8] Aartsma-Rus, A., van Ommen, G.J., Less is more: therapeutic exon skipping for Duchenne muscular dystrophy. Lancet Neurol 8 (2009), 873–875.
    • (2009) Lancet Neurol , vol.8 , pp. 873-875
    • Aartsma-Rus, A.1    van Ommen, G.J.2
  • 10
    • 80051690306 scopus 로고    scopus 로고
    • Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
    • [10] Cirak, S., Arechavala-Gomeza, V., Guglieri, M., Feng, L., Torelli, S., Anthony, K., et al. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. Lancet 378 (2011), 595–605.
    • (2011) Lancet , vol.378 , pp. 595-605
    • Cirak, S.1    Arechavala-Gomeza, V.2    Guglieri, M.3    Feng, L.4    Torelli, S.5    Anthony, K.6
  • 11
    • 84907998273 scopus 로고    scopus 로고
    • A rebirth for drisapersen in Duchenne muscular dystrophy?
    • [11] Hoffman, E., A rebirth for drisapersen in Duchenne muscular dystrophy?. Lancet Neurol 13 (2014), 963–965.
    • (2014) Lancet Neurol , vol.13 , pp. 963-965
    • Hoffman, E.1
  • 12
    • 77949512149 scopus 로고    scopus 로고
    • Identification of small molecule and genetic modulators of AON-induced dystrophin exon skipping by high-throughput screening
    • [12] O'Leary, D.A., Sharif, O., Anderson, P., Tu, B., Welch, G., Zhou, Y., et al. Identification of small molecule and genetic modulators of AON-induced dystrophin exon skipping by high-throughput screening. PLoS One, 4, 2009, e8348.
    • (2009) PLoS One , vol.4 , pp. e8348
    • O'Leary, D.A.1    Sharif, O.2    Anderson, P.3    Tu, B.4    Welch, G.5    Zhou, Y.6
  • 13
    • 77950519394 scopus 로고    scopus 로고
    • Guanine analogues enhance antisense oligonucleotide-induced exon skipping in dystrophin gene in vitro and in vivo
    • [13] Hu, Y., Wu, B., Zillmer, A., Lu, P., Benrashid, E., Wang, M., et al. Guanine analogues enhance antisense oligonucleotide-induced exon skipping in dystrophin gene in vitro and in vivo. Mol Ther 18 (2010), 812–818.
    • (2010) Mol Ther , vol.18 , pp. 812-818
    • Hu, Y.1    Wu, B.2    Zillmer, A.3    Lu, P.4    Benrashid, E.5    Wang, M.6
  • 14
    • 79955823029 scopus 로고    scopus 로고
    • Chemical treatment enhances skipping of a mutated exon in the dystrophin gene
    • [14] Nishida, A., Kataoka, N., Takeshima, Y., Yagi, M., Awano, H., Ota, M., et al. Chemical treatment enhances skipping of a mutated exon in the dystrophin gene. Nat Commun, 2, 2011, 308.
    • (2011) Nat Commun , vol.2 , pp. 308
    • Nishida, A.1    Kataoka, N.2    Takeshima, Y.3    Yagi, M.4    Awano, H.5    Ota, M.6
  • 15
    • 0027470203 scopus 로고
    • The structural and functional diversity of dystrophin
    • [15] Ahn, A.H., Kunkel, L.M., The structural and functional diversity of dystrophin. Nat Genet 3 (1993), 283–291.
    • (1993) Nat Genet , vol.3 , pp. 283-291
    • Ahn, A.H.1    Kunkel, L.M.2
  • 16
    • 34347366205 scopus 로고    scopus 로고
    • Identification of seven novel cryptic exons embedded in the dystrophin gene and characterization of 14 cryptic dystrophin exons
    • [16] Zhang, Z., Habara, Y., Nishiyama, A., Oyazato, Y., Yagi, M., Takeshima, Y., et al. Identification of seven novel cryptic exons embedded in the dystrophin gene and characterization of 14 cryptic dystrophin exons. J Hum Genet 52 (2007), 607–617.
    • (2007) J Hum Genet , vol.52 , pp. 607-617
    • Zhang, Z.1    Habara, Y.2    Nishiyama, A.3    Oyazato, Y.4    Yagi, M.5    Takeshima, Y.6
  • 17
    • 0030725216 scopus 로고    scopus 로고
    • Disruption of the splicing enhancer sequence within exon 27 of the dystrophin gene by a nonsense mutation induces partial skipping of the exon and is responsible for Becker muscular dystrophy
    • [17] Shiga, N., Takeshima, Y., Sakamoto, H., Inoue, K., Yokota, Y., Yokoyama, M., et al. Disruption of the splicing enhancer sequence within exon 27 of the dystrophin gene by a nonsense mutation induces partial skipping of the exon and is responsible for Becker muscular dystrophy. J Clin Invest 100 (1997), 2204–2210.
    • (1997) J Clin Invest , vol.100 , pp. 2204-2210
    • Shiga, N.1    Takeshima, Y.2    Sakamoto, H.3    Inoue, K.4    Yokota, Y.5    Yokoyama, M.6
  • 18
    • 34247607788 scopus 로고    scopus 로고
    • Two novel missense mutations in the myostatin gene identified in Japanese patients with Duchenne muscular dystrophy
    • [18] Nishiyama, A., Takeshima, Y., Saiki, K., Narukage, A., Oyazato, Y., Yagi, M., et al. Two novel missense mutations in the myostatin gene identified in Japanese patients with Duchenne muscular dystrophy. BMC Med Genet 8 (2007), 1–9.
    • (2007) BMC Med Genet , vol.8 , pp. 1-9
    • Nishiyama, A.1    Takeshima, Y.2    Saiki, K.3    Narukage, A.4    Oyazato, Y.5    Yagi, M.6
  • 19
    • 79951782036 scopus 로고    scopus 로고
    • Nonsense mutation-associated Becker muscular dystrophy: interplay between exon definition and splicing regulatory elements within the DMD gene
    • [19] Flanigan, K.M., Dunn, D.M., von Niederhausern, A., Soltanzadeh, P., Howard, M.T., Sampson, J.B., et al. Nonsense mutation-associated Becker muscular dystrophy: interplay between exon definition and splicing regulatory elements within the DMD gene. Hum Mutat 32 (2011), 299–308.
    • (2011) Hum Mutat , vol.32 , pp. 299-308
    • Flanigan, K.M.1    Dunn, D.M.2    von Niederhausern, A.3    Soltanzadeh, P.4    Howard, M.T.5    Sampson, J.B.6
  • 20
    • 0029114101 scopus 로고
    • Staurosporine, a potentially important gift from a microorganism
    • [20] Omura, S., Sasaki, Y., Iwai, Y., Takeshima, H., Staurosporine, a potentially important gift from a microorganism. J Antibiot (Tokyo) 48 (1995), 535–548.
    • (1995) J Antibiot (Tokyo) , vol.48 , pp. 535-548
    • Omura, S.1    Sasaki, Y.2    Iwai, Y.3    Takeshima, H.4
  • 21
    • 84887922218 scopus 로고    scopus 로고
    • Staurosporine analogues from microbial and synthetic sources and their biological activities
    • [21] Park, B.S., Abdel-Azeem, A.Z., Al-Sanea, M.M., Yoo, K.H., Tae, J.S., Lee, S.H., Staurosporine analogues from microbial and synthetic sources and their biological activities. Curr Med Chem 20 (2013), 3872–3902.
    • (2013) Curr Med Chem , vol.20 , pp. 3872-3902
    • Park, B.S.1    Abdel-Azeem, A.Z.2    Al-Sanea, M.M.3    Yoo, K.H.4    Tae, J.S.5    Lee, S.H.6
  • 22
    • 34250858877 scopus 로고    scopus 로고
    • Activation of protein phosphatase causes alternative splicing of tumor necrosis factor-related apoptosis-inducing ligand (TRAIL): potential effect on immune surveillance
    • [22] Kamachi, M., Aramaki, T., Tanimura, S., Ichinose, K., Fujikawa, K., Iwamoto, N., et al. Activation of protein phosphatase causes alternative splicing of tumor necrosis factor-related apoptosis-inducing ligand (TRAIL): potential effect on immune surveillance. Biochem Biophys Res Commun 360 (2007), 280–285.
    • (2007) Biochem Biophys Res Commun , vol.360 , pp. 280-285
    • Kamachi, M.1    Aramaki, T.2    Tanimura, S.3    Ichinose, K.4    Fujikawa, K.5    Iwamoto, N.6
  • 23
    • 84866666928 scopus 로고    scopus 로고
    • Bcl-x pre-mRNA splicing regulates brain injury after neonatal hypoxia-ischemia
    • [23] Xiao, Q., Ford, A.L., Xu, J., Yan, P., Lee, K.Y., Gonzales, E., et al. Bcl-x pre-mRNA splicing regulates brain injury after neonatal hypoxia-ischemia. J Neurosci 32 (2012), 13587–13596.
    • (2012) J Neurosci , vol.32 , pp. 13587-13596
    • Xiao, Q.1    Ford, A.L.2    Xu, J.3    Yan, P.4    Lee, K.Y.5    Gonzales, E.6
  • 24
    • 80052607346 scopus 로고    scopus 로고
    • CDK4 and cyclin D1 allow human myogenic cells to recapture growth property without compromising differentiation potential
    • [24] Shiomi, K., Kiyono, T., Okamura, K., Uezumi, M., Goto, Y., Yasumoto, S., et al. CDK4 and cyclin D1 allow human myogenic cells to recapture growth property without compromising differentiation potential. Gene Ther 18 (2011), 857–866.
    • (2011) Gene Ther , vol.18 , pp. 857-866
    • Shiomi, K.1    Kiyono, T.2    Okamura, K.3    Uezumi, M.4    Goto, Y.5    Yasumoto, S.6
  • 25
    • 40549119129 scopus 로고    scopus 로고
    • A strong exonic splicing enhancer in dystrophin exon 19 achieve proper splicing without an upstream polypyrimidine tract
    • [25] Habara, Y., Doshita, M., Hirozawa, S., Yokono, Y., Yagi, M., Takeshima, Y., et al. A strong exonic splicing enhancer in dystrophin exon 19 achieve proper splicing without an upstream polypyrimidine tract. J Biochem 143 (2008), 303–310.
    • (2008) J Biochem , vol.143 , pp. 303-310
    • Habara, Y.1    Doshita, M.2    Hirozawa, S.3    Yokono, Y.4    Yagi, M.5    Takeshima, Y.6
  • 26
    • 2342655502 scopus 로고    scopus 로고
    • Manipulation of alternative splicing by a newly developed inhibitor of CLKs
    • [26] Muraki, M., Ohkawara, B., Hosoya, T., Onogi, H., Koizumi, J., Koizumi, T., et al. Manipulation of alternative splicing by a newly developed inhibitor of CLKs. J Biol Chem 279 (2004), 24246–24254.
    • (2004) J Biol Chem , vol.279 , pp. 24246-24254
    • Muraki, M.1    Ohkawara, B.2    Hosoya, T.3    Onogi, H.4    Koizumi, J.5    Koizumi, T.6
  • 27
    • 0042671357 scopus 로고    scopus 로고
    • Pre-mRNA splicing: awash in a sea of proteins
    • [27] Jurica, M.S., Moore, M.J., Pre-mRNA splicing: awash in a sea of proteins. Mol Cell 12 (2003), 5–14.
    • (2003) Mol Cell , vol.12 , pp. 5-14
    • Jurica, M.S.1    Moore, M.J.2
  • 28
    • 60349104299 scopus 로고    scopus 로고
    • The spliceosome: design principles of a dynamic RNP machine
    • [28] Wahl, M.C., Will, C.L., Luhrmann, R., The spliceosome: design principles of a dynamic RNP machine. Cell 136 (2009), 701–718.
    • (2009) Cell , vol.136 , pp. 701-718
    • Wahl, M.C.1    Will, C.L.2    Luhrmann, R.3
  • 29
    • 38349085591 scopus 로고    scopus 로고
    • Regulation of alternative splicing by reversible protein phosphorylation
    • [29] Stamm, S., Regulation of alternative splicing by reversible protein phosphorylation. J Biol Chem 283 (2008), 1223–1227.
    • (2008) J Biol Chem , vol.283 , pp. 1223-1227
    • Stamm, S.1
  • 30
    • 39749151729 scopus 로고    scopus 로고
    • Combination of CLK family kinase and SRp75 modulates alternative splicing of Adenovirus E1A
    • [30] Yomoda, J., Muraki, M., Kataoka, N., Hosoya, T., Suzuki, M., Hagiwara, M., et al. Combination of CLK family kinase and SRp75 modulates alternative splicing of Adenovirus E1A. Genes Cells 13 (2008), 233–244.
    • (2008) Genes Cells , vol.13 , pp. 233-244
    • Yomoda, J.1    Muraki, M.2    Kataoka, N.3    Hosoya, T.4    Suzuki, M.5    Hagiwara, M.6
  • 31
    • 63649100364 scopus 로고    scopus 로고
    • Cdc2-like kinases and DNA topoisomerase I regulate alternative splicing of tissue factor in human endothelial cells
    • [31] Eisenreich, A., Bogdanov, V.Y., Zakrzewicz, A., Pries, A., Antoniak, S., Poller, W., et al. Cdc2-like kinases and DNA topoisomerase I regulate alternative splicing of tissue factor in human endothelial cells. Circ Res 104 (2009), 589–599.
    • (2009) Circ Res , vol.104 , pp. 589-599
    • Eisenreich, A.1    Bogdanov, V.Y.2    Zakrzewicz, A.3    Pries, A.4    Antoniak, S.5    Poller, W.6


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.