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New Approaches from Gene to therapies: Non-Coding Nucleic Acids

Year 2013, Volume: 4 Issue: 13, 27 - 36, 03.03.2015

Abstract

RNA interference (RNAi) also called post transcriptional gene silencing (PTGS), is a biological process in which RNA molecules inhibit gene expression, typically by causing the destruction of specific mRNA molecules. In 2006, Andrew Fire and Craig C. Mello shared the Nobel Prize in Physiology or Medicine for their work on RNA interference in the nematode worm C. elegans, which they published in 1998. It is formed of several different pathways that facilitate gene silencing and negative regulation of expression through non-coding small RNA or RNA particles like small interfering RNA (siRNA), microRNA (miRNA), ribozyme and antisense oligonucleotides. Recently, these small sequences have become a powerful tool for gene silencing in biological studies. The aim of this review is informing the readers about RNAi by explaining the mechanism of molecules which play major role in

References

  • Fire A, Xu S, Montgomery MK, Kostas SA, Driver SE, Mello CC. Potent and specific
  • genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature.
  • ; 391:806-11.
  • Paulson H, Gonzalez-Alegre P. RNAi gets its prize. Lancet Neurol. 2006; 5:997-9.
  • Kurreck, J. Antisense Technologies. Improvement through novel chemical
  • modifications. Eur J Biochem. 2003; 270:1628-1644
  • Dykxhoorn DM, Novina CD, Sharp PA. Killing the messenger: short RNAs that
  • silence gene expression. Nat Rev Mol Cell Biol. 2003; 4:457-67.
  • Tolia NH, Joshua-Tor L. Slicer and the argonautes. Nat Chem Biol. 2007;3(1):36-43.
  • Grishok A, Pasquinelli AE, Conte D, Li N, Parrish S, Ha I, Baillie DL, Fire A, Ruvkun
  • G, Mello CC.Genes and mechanisms related to RNA interference regulate expression
  • of the small temporal RNAs that control C elegans developmental timing. Cell. 2001;
  • :23-34.
  • Mello CC, Conte D Jr. Revealing the world of RNA interference. Nature. 2004;
  • :338-42.
  • Lee YS, Dutta A. MicroRNAs in cancer. Annu Rev Pathol Mech Dis. 2009; 4:199-
  • -
  • Zeng Y, Yi R, Cullen BR. MicroRNAs and small interfering RNAs can inhibit mRNA
  • expression by similar mechanisms. Proc Natl Acad Sci USA. 2003; 100:5779-84.
  • Hammond SM, Bernstein E, Beach D, Hannon GJ. An RNA-directed nuclease
  • mediates post-transcriptional gene silencing in Drosophila cells. Nature. 2000;
  • :293-6.
  • Zamore PD, Tuschl T, Sharp PA, Bartel DP. RNAi: double-stranded RNA directs the
  • ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals. Cell. 2000;
  • :25-33.
  • http://www.pulmonarycirculation.org/viewimage.asp?img=PulmCirc_2011_1_3_357_
  • _f2.jpg 05.12.2013.
  • Caudy AA, Myers M, Hannon GJ, Hammond SM. Fragile X-related protein and VIG
  • associate with the RNA interference machinery. Genes Dev. 2002; 16:2491-6.
  • Hamilton AJ, Baulcombe DC. A species of small antisense RNA in posttranscriptional
  • gene silencing in plants. Science. 1999; 286(5441):950–2.
  • Aigner A. Gene silencing through RNA interference (RNAi) in vivo: strategies based
  • on the direct application of siRNAs. J Biotechnol. 2006; 124(1):12-25.
  • Cowland JB, Hother C, Gronbaek K. MicroRNAs and cancer. APMIS. 2007;
  • :1090-106.
  • Krützfeldt J, Rajewsky N, Braich R, Rajeev KG, Tuschl T, Manoharan M, Stoffel M.
  • Silencing of microRNAs in vivo with ‘antagomirs’. Nature. 2005; 438:685-9.
  • Morris KV, Rossi JJ. Lentiviral-mediated delivery of siRNAs for antiviral therapy.
  • Gene Ther. 2006; 13:553-8.
  • Grimm D, Kay MA. Therapeutic short hairpin RNA expression in the liver: viral
  • targets and vectors. Gene Ther. 2006; 13:563-75.
  • Kim DH, Rossi JJ. Strategies for silencing human disease using RNA interference.
  • Nature Rev Genet. 2007; 8:173-84.
  • Ozvaran MK. Malign Mezotelyomada Gen Tedavisi. Toraks Dergisi, 2004; 5(2):110-
  • -
  • Juliano RL, Dixit VR, Kang H, Kim TY, Miyamoto Y, Xu D. Epigenetic manipulation
  • of gene expression: a toolkit for cell biologists. J Cell Biol. 2005; 169:847–857.
  • Zamecnik PC, Stephanson ML. Inhibition of Raus sarcoma virus replication and cell
  • transformation by a spesific oligodeoxynucleotide. 1978;75:280-4.
  • Zamaratski E, Pradeepkumar PI, Chattopadhyaya J. A critical survey of the structurefunction
  • of the antisense oligo/RNA heteroduplex as substrate for RNase H. J
  • Biochem Biophys Methods. 2001; 48:189–208.
  • Maeda Y, Sheffield AM, Smith RJ.Therapeutic regulation of gene expression in
  • the inner ear using RNA interference.Adv Otorhinolaryngol. 2009; 66:13-36.
  • Dass CR. Liposome-mediated delivery of oligodeoxynucleotides in vivo. Drug Deliv.
  • ; 9:169–180.
  • Lilley DM. Structure, folding and mechanisms of ribozymes. Curr. Opin. Struct. Biol.
  • ; 15:313-323.
  • Serganov A, Patel DJ. Ribozymes, riboswitches and beyond: regulation of gene
  • expression without proteins. Nat Rev Genet. 2007; 8:776-790.
  • Kruger K, Grabowski PJ, Zaug AJ. Sands J, Gottschling DE, Cech TR. Self-splicing
  • RNA: autoexcision and autocyclization of the ribosomal RNA intervening sequence of
  • Tetrahymena. Cell. 1982; 31:147-157.
  • Chen X, Li N, Ellington AD. Ribozyme catalysis of metabolism in the RNA world.
  • Chem Biodivers. 2007; 4:633-655.
  • Strobel SA, Cochrane JC. RNA catalysis: ribozymes, ribosomes, and riboswitches.
  • Curr. Opin. Chem. Biol. 2007; 11:636-643.
  • Opalinska JB, Gewirtz AM. Nucleic-acid therapeutics: basic principles and recent
  • applications. Nat. Rev. Drug Discov. 2002; 1:503-514.
  • Kore AR, Vaish NK, Kutzke U, Eckstein F. Sequence specificity of the hammerhead
  • ribozyme revisited; the NHH rule. Nucleic Acids Res. 1998; 26:4116–4120.
  • Akashi H, Matsumoto S, Taira K. Gene discovery by ribozyme and siRNA libraries.
  • Nature Reviews Molecular Cell Biology. 2005; 6(5):413-22.
  • Karaboz I, Çolak C. Antisens Teknolojisi. Orlab On-Line Mikrobiyoloji Dergisi. 2007;
  • (2):14-37.

GENDEN TEDAVİYE YENİ YAKLAŞIMLAR: KODLAMAYAN NÜKLEİK ASİTLER

Year 2013, Volume: 4 Issue: 13, 27 - 36, 03.03.2015

Abstract

Posttranskripsiyonel (transkripsiyon sonrası) gen susturma (PTGS) olarak da adlandırılan RNA interferans, RNA molekülünün, spesifik mRNA molekülünün yıkımına sebep olarak, gen ekspresyonunu inhibe ettiği biyolojik bir süreçtir. 2006 yılında, Andrew Fire ve Craig Mello, Caenorhabditis elegans nematodunda ‘Ribonükleik asit interferansı’ (RNAi) üzerine yaptıkları çalışma ile(1998 yılında yayınlanan) Fizyoloji ve Tıp dalında Nobel ödülüne layık görüldüler. Gen ifadesinin susturulmasını sağlayan RNAi yolakları, small interfering RNA (siRNA), mikro RNA (miRNA), ribozim ve antisens oligonükleotitler adı verilen küçük, protein kodlamayan ve gen ifadesinin negatif düzenleyicisi olan RNA ve DNA parçacıklarının aracılığıyla gerçekleşmektedir. Son yıllarda, bu küçük RNA ve DNA dizileri biyolojik araştırmalarda gen susturulması için kullanılan çok güçlü bir yöntem haline gelmiştir. Bu derlemede, RNAi yolağında kilit rol oynayan moleküllerin mekanizmasına değinerek RNAi hakkında okuyucunun bilgilendirilmesi amaçlanmaktadır.

References

  • Fire A, Xu S, Montgomery MK, Kostas SA, Driver SE, Mello CC. Potent and specific
  • genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature.
  • ; 391:806-11.
  • Paulson H, Gonzalez-Alegre P. RNAi gets its prize. Lancet Neurol. 2006; 5:997-9.
  • Kurreck, J. Antisense Technologies. Improvement through novel chemical
  • modifications. Eur J Biochem. 2003; 270:1628-1644
  • Dykxhoorn DM, Novina CD, Sharp PA. Killing the messenger: short RNAs that
  • silence gene expression. Nat Rev Mol Cell Biol. 2003; 4:457-67.
  • Tolia NH, Joshua-Tor L. Slicer and the argonautes. Nat Chem Biol. 2007;3(1):36-43.
  • Grishok A, Pasquinelli AE, Conte D, Li N, Parrish S, Ha I, Baillie DL, Fire A, Ruvkun
  • G, Mello CC.Genes and mechanisms related to RNA interference regulate expression
  • of the small temporal RNAs that control C elegans developmental timing. Cell. 2001;
  • :23-34.
  • Mello CC, Conte D Jr. Revealing the world of RNA interference. Nature. 2004;
  • :338-42.
  • Lee YS, Dutta A. MicroRNAs in cancer. Annu Rev Pathol Mech Dis. 2009; 4:199-
  • -
  • Zeng Y, Yi R, Cullen BR. MicroRNAs and small interfering RNAs can inhibit mRNA
  • expression by similar mechanisms. Proc Natl Acad Sci USA. 2003; 100:5779-84.
  • Hammond SM, Bernstein E, Beach D, Hannon GJ. An RNA-directed nuclease
  • mediates post-transcriptional gene silencing in Drosophila cells. Nature. 2000;
  • :293-6.
  • Zamore PD, Tuschl T, Sharp PA, Bartel DP. RNAi: double-stranded RNA directs the
  • ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals. Cell. 2000;
  • :25-33.
  • http://www.pulmonarycirculation.org/viewimage.asp?img=PulmCirc_2011_1_3_357_
  • _f2.jpg 05.12.2013.
  • Caudy AA, Myers M, Hannon GJ, Hammond SM. Fragile X-related protein and VIG
  • associate with the RNA interference machinery. Genes Dev. 2002; 16:2491-6.
  • Hamilton AJ, Baulcombe DC. A species of small antisense RNA in posttranscriptional
  • gene silencing in plants. Science. 1999; 286(5441):950–2.
  • Aigner A. Gene silencing through RNA interference (RNAi) in vivo: strategies based
  • on the direct application of siRNAs. J Biotechnol. 2006; 124(1):12-25.
  • Cowland JB, Hother C, Gronbaek K. MicroRNAs and cancer. APMIS. 2007;
  • :1090-106.
  • Krützfeldt J, Rajewsky N, Braich R, Rajeev KG, Tuschl T, Manoharan M, Stoffel M.
  • Silencing of microRNAs in vivo with ‘antagomirs’. Nature. 2005; 438:685-9.
  • Morris KV, Rossi JJ. Lentiviral-mediated delivery of siRNAs for antiviral therapy.
  • Gene Ther. 2006; 13:553-8.
  • Grimm D, Kay MA. Therapeutic short hairpin RNA expression in the liver: viral
  • targets and vectors. Gene Ther. 2006; 13:563-75.
  • Kim DH, Rossi JJ. Strategies for silencing human disease using RNA interference.
  • Nature Rev Genet. 2007; 8:173-84.
  • Ozvaran MK. Malign Mezotelyomada Gen Tedavisi. Toraks Dergisi, 2004; 5(2):110-
  • -
  • Juliano RL, Dixit VR, Kang H, Kim TY, Miyamoto Y, Xu D. Epigenetic manipulation
  • of gene expression: a toolkit for cell biologists. J Cell Biol. 2005; 169:847–857.
  • Zamecnik PC, Stephanson ML. Inhibition of Raus sarcoma virus replication and cell
  • transformation by a spesific oligodeoxynucleotide. 1978;75:280-4.
  • Zamaratski E, Pradeepkumar PI, Chattopadhyaya J. A critical survey of the structurefunction
  • of the antisense oligo/RNA heteroduplex as substrate for RNase H. J
  • Biochem Biophys Methods. 2001; 48:189–208.
  • Maeda Y, Sheffield AM, Smith RJ.Therapeutic regulation of gene expression in
  • the inner ear using RNA interference.Adv Otorhinolaryngol. 2009; 66:13-36.
  • Dass CR. Liposome-mediated delivery of oligodeoxynucleotides in vivo. Drug Deliv.
  • ; 9:169–180.
  • Lilley DM. Structure, folding and mechanisms of ribozymes. Curr. Opin. Struct. Biol.
  • ; 15:313-323.
  • Serganov A, Patel DJ. Ribozymes, riboswitches and beyond: regulation of gene
  • expression without proteins. Nat Rev Genet. 2007; 8:776-790.
  • Kruger K, Grabowski PJ, Zaug AJ. Sands J, Gottschling DE, Cech TR. Self-splicing
  • RNA: autoexcision and autocyclization of the ribosomal RNA intervening sequence of
  • Tetrahymena. Cell. 1982; 31:147-157.
  • Chen X, Li N, Ellington AD. Ribozyme catalysis of metabolism in the RNA world.
  • Chem Biodivers. 2007; 4:633-655.
  • Strobel SA, Cochrane JC. RNA catalysis: ribozymes, ribosomes, and riboswitches.
  • Curr. Opin. Chem. Biol. 2007; 11:636-643.
  • Opalinska JB, Gewirtz AM. Nucleic-acid therapeutics: basic principles and recent
  • applications. Nat. Rev. Drug Discov. 2002; 1:503-514.
  • Kore AR, Vaish NK, Kutzke U, Eckstein F. Sequence specificity of the hammerhead
  • ribozyme revisited; the NHH rule. Nucleic Acids Res. 1998; 26:4116–4120.
  • Akashi H, Matsumoto S, Taira K. Gene discovery by ribozyme and siRNA libraries.
  • Nature Reviews Molecular Cell Biology. 2005; 6(5):413-22.
  • Karaboz I, Çolak C. Antisens Teknolojisi. Orlab On-Line Mikrobiyoloji Dergisi. 2007;
  • (2):14-37.
There are 75 citations in total.

Details

Primary Language Turkish
Subjects Health Care Administration
Journal Section Review
Authors

Hasret Ecevit This is me

Sedat Motor This is me

Müzeyyen İzmirli This is me

Publication Date March 3, 2015
Submission Date March 1, 2015
Published in Issue Year 2013 Volume: 4 Issue: 13

Cite

Vancouver Ecevit H, Motor S, İzmirli M. GENDEN TEDAVİYE YENİ YAKLAŞIMLAR: KODLAMAYAN NÜKLEİK ASİTLER. mkutfd. 2015;4(13):27-36.