메뉴 건너뛰기




Volumn 76, Issue , 2016, Pages 408-417

Method of carrier-free delivery of therapeutic RNA importable into human mitochondria: Lipophilic conjugates with cleavable bonds

Author keywords

Antireplicative RNA; Cell delivery; Mitochondrial diseases; Mitochondrial drug delivery; RNA conjugates; RNA therapeutics

Indexed keywords

CELLS; CHOLESTEROL; CYTOLOGY; DNA; GENE THERAPY; MOLECULAR BIOLOGY; NUCLEIC ACIDS; OLIGONUCLEOTIDES; RNA;

EID: 84961233517     PISSN: 01429612     EISSN: 18785905     Source Type: Journal    
DOI: 10.1016/j.biomaterials.2015.10.075     Document Type: Article
Times cited : (31)

References (45)
  • 1
    • 84856389509 scopus 로고    scopus 로고
    • RNA-based therapeutics: current progress and future prospects
    • Burnett J.C., Rossi J.J. RNA-based therapeutics: current progress and future prospects. Chem. Biol. 2012, 19:60-71.
    • (2012) Chem. Biol. , vol.19 , pp. 60-71
    • Burnett, J.C.1    Rossi, J.J.2
  • 3
    • 77951153670 scopus 로고    scopus 로고
    • Selection of RNA aptamers imported into yeast and human mitochondria
    • Kolesnikova O., et al. Selection of RNA aptamers imported into yeast and human mitochondria. RNA 2010, 16:926-941.
    • (2010) RNA , vol.16 , pp. 926-941
    • Kolesnikova, O.1
  • 4
    • 84871750408 scopus 로고    scopus 로고
    • Mitochondrial targeting of recombinant RNAs modulates the level of a heteroplasmic mutation in human mitochondrial DNA associated with Kearns Sayre Syndrome
    • Comte C., et al. Mitochondrial targeting of recombinant RNAs modulates the level of a heteroplasmic mutation in human mitochondrial DNA associated with Kearns Sayre Syndrome. Nucleic Acids Res. 2013, 41:418-433.
    • (2013) Nucleic Acids Res. , vol.41 , pp. 418-433
    • Comte, C.1
  • 5
    • 33846094306 scopus 로고    scopus 로고
    • An enhanced MITOMAP with a global mtDNA mutational phylogeny
    • Ruiz-Pesini E., et al. An enhanced MITOMAP with a global mtDNA mutational phylogeny. Nucleic Acids Res. 2007, 35:D823-D828.
    • (2007) Nucleic Acids Res. , vol.35 , pp. D823-D828
    • Ruiz-Pesini, E.1
  • 6
    • 77953507107 scopus 로고    scopus 로고
    • Mitochondrial DNA mutations in disease and aging
    • Wallace D.C. Mitochondrial DNA mutations in disease and aging. Environ. Mol. Mutagen. 2010, 51:440-450.
    • (2010) Environ. Mol. Mutagen. , vol.51 , pp. 440-450
    • Wallace, D.C.1
  • 7
    • 17744393686 scopus 로고    scopus 로고
    • Mitochondrial DNA mutations in human disease
    • Taylor R.W., Turnbull D.M. Mitochondrial DNA mutations in human disease. Nat. Rev. Genet. 2005, 6:389-402.
    • (2005) Nat. Rev. Genet. , vol.6 , pp. 389-402
    • Taylor, R.W.1    Turnbull, D.M.2
  • 8
    • 84900401998 scopus 로고    scopus 로고
    • Modeling of antigenomic therapy of mitochondrial diseases by mitochondrially addressed RNA targeting a pathogenic point mutation in mitochondrial DNA
    • Tonin Y., et al. Modeling of antigenomic therapy of mitochondrial diseases by mitochondrially addressed RNA targeting a pathogenic point mutation in mitochondrial DNA. J. Biol. Chem. 2014, 289:13323-13334.
    • (2014) J. Biol. Chem. , vol.289 , pp. 13323-13334
    • Tonin, Y.1
  • 9
    • 84872863485 scopus 로고    scopus 로고
    • Nanoparticle-based delivery of RNAi therapeutics: progress and challenges
    • Zhou J., Shum K.T., Burnett J.C., Rossi J.J. Nanoparticle-based delivery of RNAi therapeutics: progress and challenges. Pharm. - Basel Switz. 2013, 6:85-107.
    • (2013) Pharm. - Basel Switz. , vol.6 , pp. 85-107
    • Zhou, J.1    Shum, K.T.2    Burnett, J.C.3    Rossi, J.J.4
  • 10
    • 33746438424 scopus 로고    scopus 로고
    • Toxicity of cationic lipids and cationic polymers in gene delivery
    • Lv H., Zhang S., Wang B., Cui S., Yan J. Toxicity of cationic lipids and cationic polymers in gene delivery. J. Control Release 2006, 114:100-109.
    • (2006) J. Control Release , vol.114 , pp. 100-109
    • Lv, H.1    Zhang, S.2    Wang, B.3    Cui, S.4    Yan, J.5
  • 11
    • 84880915038 scopus 로고    scopus 로고
    • Oligonucleotide conjugates for therapeutic applications
    • Winkler J. Oligonucleotide conjugates for therapeutic applications. Ther. Deliv. 2013, 4:791-809.
    • (2013) Ther. Deliv. , vol.4 , pp. 791-809
    • Winkler, J.1
  • 12
    • 35148815670 scopus 로고    scopus 로고
    • Mechanisms and optimization of in vivo delivery of lipophilic siRNAs
    • Wolfrum C., et al. Mechanisms and optimization of in vivo delivery of lipophilic siRNAs. Nat. Biotechnol. 2007, 25:1149-1157.
    • (2007) Nat. Biotechnol. , vol.25 , pp. 1149-1157
    • Wolfrum, C.1
  • 13
    • 84858396767 scopus 로고    scopus 로고
    • Carrier-free cellular uptake and the gene-silencing activity of the lipophilic siRNAs is strongly affected by the length of the linker between siRNA and lipophilic group
    • Petrova N.S., et al. Carrier-free cellular uptake and the gene-silencing activity of the lipophilic siRNAs is strongly affected by the length of the linker between siRNA and lipophilic group. Nucleic Acids Res. 2012, 40:2330-2344.
    • (2012) Nucleic Acids Res. , vol.40 , pp. 2330-2344
    • Petrova, N.S.1
  • 14
    • 0000800447 scopus 로고
    • Cholesteryl-conjugated oligonucleotides: synthesis, properties, and activity as inhibitors of replication of human immunodeficiency virus in cell culture
    • Letsinger R.L., Zhang G.R., Sun D.K., Ikeuchi T., Sarin P.S. Cholesteryl-conjugated oligonucleotides: synthesis, properties, and activity as inhibitors of replication of human immunodeficiency virus in cell culture. Proc. Natl. Acad. Sci. U. S. A. 1989, 86:6553-6556.
    • (1989) Proc. Natl. Acad. Sci. U. S. A. , vol.86 , pp. 6553-6556
    • Letsinger, R.L.1    Zhang, G.R.2    Sun, D.K.3    Ikeuchi, T.4    Sarin, P.S.5
  • 15
    • 24144489048 scopus 로고    scopus 로고
    • Reversible covalent chemistry in drug delivery
    • West K.R., Otto S. Reversible covalent chemistry in drug delivery. Curr. Drug Discov. Technol. 2005, 2:123-160.
    • (2005) Curr. Drug Discov. Technol. , vol.2 , pp. 123-160
    • West, K.R.1    Otto, S.2
  • 16
    • 84977942201 scopus 로고    scopus 로고
    • Duplex oligonucleotide complexes and methods for gene silencing by RNA interference
    • C. Yamada, A. Khvorova, R. Kaiser, E. Anderson, D. Leake. Duplex oligonucleotide complexes and methods for gene silencing by RNA interference. Google Patents, (2013).
    • (2013) Google Patents
    • Yamada, C.1    Khvorova, A.2    Kaiser, R.3    Anderson, E.4    Leake, D.5
  • 17
    • 84861569173 scopus 로고    scopus 로고
    • Structure-activity relationships and mechanism of action of Eph-ephrin antagonists: interaction of cholanic acid with the EphA2 receptor
    • Tognolini M., et al. Structure-activity relationships and mechanism of action of Eph-ephrin antagonists: interaction of cholanic acid with the EphA2 receptor. ChemMedChem 2012, 7:1071-1083.
    • (2012) ChemMedChem , vol.7 , pp. 1071-1083
    • Tognolini, M.1
  • 18
    • 33749529741 scopus 로고    scopus 로고
    • Synthesis of poly(beta-amino ester)s with thiol-reactive side chains for DNA delivery
    • Zugates G.T., Anderson D.G., Little S.R., Lawhorn I.E., Langer R. Synthesis of poly(beta-amino ester)s with thiol-reactive side chains for DNA delivery. J. Am. Chem. Soc. 2006, 128:12726-12734.
    • (2006) J. Am. Chem. Soc. , vol.128 , pp. 12726-12734
    • Zugates, G.T.1    Anderson, D.G.2    Little, S.R.3    Lawhorn, I.E.4    Langer, R.5
  • 19
    • 0034808094 scopus 로고    scopus 로고
    • The structural role of cholesterol in biological membranes
    • Sugahara M., Uragami M., Yan X., Regen S.L. The structural role of cholesterol in biological membranes. J. Am. Chem. Soc. 2001, 123:7939-7940.
    • (2001) J. Am. Chem. Soc. , vol.123 , pp. 7939-7940
    • Sugahara, M.1    Uragami, M.2    Yan, X.3    Regen, S.L.4
  • 20
    • 43349093551 scopus 로고    scopus 로고
    • Oligoribonucleotides with 2'-O-(tert-butyldimethylsilyl) groups
    • Chapter 3: Unit 3 6
    • Bellon L. Oligoribonucleotides with 2'-O-(tert-butyldimethylsilyl) groups. Curr. Protoc. Nucleic Acid Chem. 2001, Chapter 3: Unit 3 6.
    • (2001) Curr. Protoc. Nucleic Acid Chem.
    • Bellon, L.1
  • 21
    • 84926636581 scopus 로고    scopus 로고
    • Mitochondrial targeting of recombinant RNA
    • Dovydenko I., et al. Mitochondrial targeting of recombinant RNA. Methods Mol. Biol. 2015, 1265:209-225.
    • (2015) Methods Mol. Biol. , vol.1265 , pp. 209-225
    • Dovydenko, I.1
  • 23
    • 84895436450 scopus 로고    scopus 로고
    • Segmentation and quantification of subcellular structures in fluorescence microscopy images using Squassh
    • Rizk A., et al. Segmentation and quantification of subcellular structures in fluorescence microscopy images using Squassh. Nat. Protoc. 2014, 9:586-596.
    • (2014) Nat. Protoc. , vol.9 , pp. 586-596
    • Rizk, A.1
  • 24
    • 84879151792 scopus 로고    scopus 로고
    • Induced tRNA import into human mitochondria: implication of a host aminoacyl-tRNA-synthetase
    • Gowher A., Smirnov A., Tarassov I., Entelis N. Induced tRNA import into human mitochondria: implication of a host aminoacyl-tRNA-synthetase. PLoS One 2013, 8:e66228.
    • (2013) PLoS One , vol.8 , pp. e66228
    • Gowher, A.1    Smirnov, A.2    Tarassov, I.3    Entelis, N.4
  • 25
    • 0026698060 scopus 로고
    • Oxidized redox state of glutathione in the endoplasmic reticulum
    • Hwang C., Sinskey A.J., Lodish H.F. Oxidized redox state of glutathione in the endoplasmic reticulum. Science 1992, 257:1496-1502.
    • (1992) Science , vol.257 , pp. 1496-1502
    • Hwang, C.1    Sinskey, A.J.2    Lodish, H.F.3
  • 26
    • 81355143220 scopus 로고    scopus 로고
    • Liposomes for use in gene delivery
    • Balazs D.A., Godbey W. Liposomes for use in gene delivery. J. Drug Deliv. 2011, 2011:326497.
    • (2011) J. Drug Deliv. , vol.2011 , pp. 326497
    • Balazs, D.A.1    Godbey, W.2
  • 27
    • 4644330955 scopus 로고    scopus 로고
    • Toll-like receptor 9 binds single-stranded CpG-DNA in a sequence- and pH-dependent manner
    • Rutz M., et al. Toll-like receptor 9 binds single-stranded CpG-DNA in a sequence- and pH-dependent manner. Eur. J. Immunol. 2004, 34:2541-2550.
    • (2004) Eur. J. Immunol. , vol.34 , pp. 2541-2550
    • Rutz, M.1
  • 28
    • 39549117983 scopus 로고    scopus 로고
    • Delivery vehicles for small interfering RNA in vivo
    • de Fougerolles A.R. Delivery vehicles for small interfering RNA in vivo. Hum. Gene Ther. 2008, 19:125-132.
    • (2008) Hum. Gene Ther. , vol.19 , pp. 125-132
    • de Fougerolles, A.R.1
  • 29
    • 84905506792 scopus 로고    scopus 로고
    • Enhancing siRNA delivery by employing lipid nanoparticles
    • Hope M.J. Enhancing siRNA delivery by employing lipid nanoparticles. Ther. Deliv. 2014, 5:663-673.
    • (2014) Ther. Deliv. , vol.5 , pp. 663-673
    • Hope, M.J.1
  • 30
    • 84920918393 scopus 로고    scopus 로고
    • Efficient delivery of RNAi prodrugs containing reversible charge-neutralizing phosphotriester backbone modifications
    • Meade B.R., et al. Efficient delivery of RNAi prodrugs containing reversible charge-neutralizing phosphotriester backbone modifications. Nat. Biotechnol. 2014, 32:1256-1261.
    • (2014) Nat. Biotechnol. , vol.32 , pp. 1256-1261
    • Meade, B.R.1
  • 32
    • 81855211988 scopus 로고    scopus 로고
    • Early steps in steroidogenesis: intracellular cholesterol trafficking
    • Miller W.L., Bose H.S. Early steps in steroidogenesis: intracellular cholesterol trafficking. J. Lipid Res. 2011, 52:2111-2135.
    • (2011) J. Lipid Res. , vol.52 , pp. 2111-2135
    • Miller, W.L.1    Bose, H.S.2
  • 33
    • 84896998596 scopus 로고    scopus 로고
    • Structure of the mitochondrial translocator protein in complex with a diagnostic ligand
    • Jaremko L., Jaremko M., Giller K., Becker S., Zweckstetter M. Structure of the mitochondrial translocator protein in complex with a diagnostic ligand. Science 2014, 343:1363-1366.
    • (2014) Science , vol.343 , pp. 1363-1366
    • Jaremko, L.1    Jaremko, M.2    Giller, K.3    Becker, S.4    Zweckstetter, M.5
  • 35
    • 77952675911 scopus 로고    scopus 로고
    • Lipophilic siRNAs mediate efficient gene silencing in oligodendrocytes with direct CNS delivery
    • Chen Q., et al. Lipophilic siRNAs mediate efficient gene silencing in oligodendrocytes with direct CNS delivery. J. Control Release 2010, 144:227-232.
    • (2010) J. Control Release , vol.144 , pp. 227-232
    • Chen, Q.1
  • 36
    • 84893327896 scopus 로고    scopus 로고
    • Programmed assembly of polymer-DNA conjugate nanoparticles with optical readout and sequence-specific activation of biorecognition
    • Magnusson J.P., Fernandez-Trillo F., Sicilia G., Spain S.G., Alexander C. Programmed assembly of polymer-DNA conjugate nanoparticles with optical readout and sequence-specific activation of biorecognition. Nanoscale 2014, 6:2368-2374.
    • (2014) Nanoscale , vol.6 , pp. 2368-2374
    • Magnusson, J.P.1    Fernandez-Trillo, F.2    Sicilia, G.3    Spain, S.G.4    Alexander, C.5
  • 38
    • 84907267817 scopus 로고    scopus 로고
    • Progressive increase in mtDNA 3243A>G heteroplasmy causes abrupt transcriptional reprogramming
    • Picard M., et al. Progressive increase in mtDNA 3243A>G heteroplasmy causes abrupt transcriptional reprogramming. Proc. Natl. Acad. Sci. U. S. A. 2014, 111:E4033-E4042.
    • (2014) Proc. Natl. Acad. Sci. U. S. A. , vol.111 , pp. E4033-E4042
    • Picard, M.1
  • 39
    • 73949119072 scopus 로고    scopus 로고
    • Pearson syndrome in the neonatal period: two case reports and review of the literature
    • Manea E.M., et al. Pearson syndrome in the neonatal period: two case reports and review of the literature. J. Pediatr. Hematol. Oncol. 2009, 31:947-951.
    • (2009) J. Pediatr. Hematol. Oncol. , vol.31 , pp. 947-951
    • Manea, E.M.1
  • 40
    • 67649994021 scopus 로고    scopus 로고
    • Transmitochondrial embryonic stem cells containing pathogenic mtDNA mutations are compromised in neuronal differentiation
    • Kirby D.M., et al. Transmitochondrial embryonic stem cells containing pathogenic mtDNA mutations are compromised in neuronal differentiation. Cell Prolif. 2009, 42:413-424.
    • (2009) Cell Prolif. , vol.42 , pp. 413-424
    • Kirby, D.M.1
  • 41
    • 84946567214 scopus 로고    scopus 로고
    • Mouse somatic mutation orthologous to MELAS A3302G mutation in the mitochondrial tRNA gene confers respiration defects
    • Epub ahead of print)
    • Shimizu A., et al. Mouse somatic mutation orthologous to MELAS A3302G mutation in the mitochondrial tRNA gene confers respiration defects. Biochem. Biophys. Res. Commun. 2015 Sep 14, pii: S0006-291X(15)30589-1. (Epub ahead of print). 10.1016/j.bbrc.2015.09.072.
    • (2015) Biochem. Biophys. Res. Commun.
    • Shimizu, A.1
  • 42
    • 84928395483 scopus 로고    scopus 로고
    • Selective elimination of mitochondrial mutations in the germline by genome editing
    • Reddy P., et al. Selective elimination of mitochondrial mutations in the germline by genome editing. Cell 2015, 161:459-469.
    • (2015) Cell , vol.161 , pp. 459-469
    • Reddy, P.1
  • 43
    • 78650486114 scopus 로고    scopus 로고
    • Segregation of mtDNA throughout human embryofetal development: m.3243A>G as a model system
    • Monnot S., et al. Segregation of mtDNA throughout human embryofetal development: m.3243A>G as a model system. Hum. Mutat. 2011, 32:116-125.
    • (2011) Hum. Mutat. , vol.32 , pp. 116-125
    • Monnot, S.1
  • 44
    • 39349105943 scopus 로고    scopus 로고
    • A mouse model of mitochondrial disease reveals germline selection against severe mtDNA mutations
    • Fan W., et al. A mouse model of mitochondrial disease reveals germline selection against severe mtDNA mutations. Science 2008, 319:958-962.
    • (2008) Science , vol.319 , pp. 958-962
    • Fan, W.1
  • 45
    • 80155155905 scopus 로고    scopus 로고
    • Transmitochondrial mice as models for mitochondrial DNA-based diseases
    • Nakada K., Hayashi J. Transmitochondrial mice as models for mitochondrial DNA-based diseases. Exp. Anim. 2011, 60:421-431.
    • (2011) Exp. Anim. , vol.60 , pp. 421-431
    • Nakada, K.1    Hayashi, J.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.