RNA interference: potential therapeutic targets

One of the most exciting findings in recent years has been the discovery of RNA interference (RNAi). RNAi methodologies hold the promise to selectively inhibit gene expression in mammals. RNAi is an innate cellular process activated when a double-stranded RNA (dsRNA) molecule of greater than 19 duplex nucleotides enters the cell, causing the degradation of not only the invading dsRNA molecule, but also single-stranded (ssRNAs) RNAs of identical sequences, including endogenous mRNAs. The use of RNAi for genetic-based therapies has been widely studied, especially in viral infections, cancers, and inherited genetic disorders. As such, RNAi technology is a potentially useful method to develop highly specific dsRNA-based gene-silencing therapeutics.

[1]  C. Napoli,et al.  Introduction of a Chimeric Chalcone Synthase Gene into Petunia Results in Reversible Co-Suppression of Homologous Genes in trans. , 1990, The Plant cell.

[2]  V. Ambros,et al.  The C. elegans heterochronic gene lin-4 encodes small RNAs with antisense complementarity to lin-14 , 1993, Cell.

[3]  K. Kemphues,et al.  par-1, a gene required for establishing polarity in C. elegans embryos, encodes a putative Ser/Thr kinase that is asymmetrically distributed , 1995, Cell.

[4]  G. Wang,et al.  Induction of the , 1996 .

[5]  R. Kucherlapati,et al.  K-ras is an essential gene in the mouse with partial functional overlap with N-ras. , 1997, Genes & development.

[6]  Jun Miyoshi,et al.  K-Ras is essential for the development of the mouse embryo , 1997, Oncogene.

[7]  A. Fire,et al.  Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans , 1998, Nature.

[8]  C. Mello,et al.  RNAi in C. elegans: Soaking in the Genome Sequence , 1998, Science.

[9]  R. Plasterk,et al.  mut-7 of C. elegans, Required for Transposon Silencing and RNA Interference, Is a Homolog of Werner Syndrome Helicase and RNaseD , 1999, Cell.

[10]  Andrew Fire,et al.  The rde-1 Gene, RNA Interference, and Transposon Silencing in C. elegans , 1999, Cell.

[11]  Magdalena Zernicka-Goetz,et al.  Specific interference with gene function by double-stranded RNA in early mouse development , 2000, Nature Cell Biology.

[12]  K. Ui-Tei,et al.  Sensitive assay of RNA interference in Drosophila and Chinese hamster cultured cells using firefly luciferase gene as target , 2000, FEBS letters.

[13]  S. Hammond,et al.  An RNA-directed nuclease mediates post-transcriptional gene silencing in Drosophila cells , 2000, Nature.

[14]  Ronald H. A. Plasterk,et al.  A genetic link between co-suppression and RNA interference in C. elegans , 2000, Nature.

[15]  R. Carthew,et al.  Heritable gene silencing in Drosophila using double-stranded RNA , 2000, Nature Biotechnology.

[16]  P. Sharp,et al.  RNAi Double-Stranded RNA Directs the ATP-Dependent Cleavage of mRNA at 21 to 23 Nucleotide Intervals , 2000, Cell.

[17]  B. Reinhart,et al.  The 21-nucleotide let-7 RNA regulates developmental timing in Caenorhabditis elegans , 2000, Nature.

[18]  Hagstrom Je Self-assembling complexes for in vivo gene delivery. , 2000 .

[19]  T. Tuschl,et al.  RNA interference is mediated by 21- and 22-nucleotide RNAs. , 2001, Genes & development.

[20]  V. Ambros,et al.  An Extensive Class of Small RNAs in Caenorhabditis elegans , 2001, Science.

[21]  A. Pasquinelli,et al.  A Cellular Function for the RNA-Interference Enzyme Dicer in the Maturation of the let-7 Small Temporal RNA , 2001, Science.

[22]  B. Kaang,et al.  Overexpression of and RNA interference with the CCAAT enhancer-binding protein on long-term facilitation of Aplysia sensory to motor synapses. , 2001, Learning & memory.

[23]  B. Bass,et al.  A Role for the RNase III Enzyme DCR-1 in RNA Interference and Germ Line Development in Caenorhabditis elegans , 2001, Science.

[24]  Debra Robertson,et al.  US FTC probes practices of 100 companies , 2001, Nature Biotechnology.

[25]  G. Hannon,et al.  C . elegans involved in developmental timing in Dicer functions in RNA interference and in synthesis of small RNA , 2001 .

[26]  A. Fire,et al.  Specific inhibition of gene expression by small double-stranded RNAs in invertebrate and vertebrate systems , 2001, Proceedings of the National Academy of Sciences of the United States of America.

[27]  A. Caudy,et al.  Argonaute2, a Link Between Genetic and Biochemical Analyses of RNAi , 2001, Science.

[28]  L. Lim,et al.  An Abundant Class of Tiny RNAs with Probable Regulatory Roles in Caenorhabditis elegans , 2001, Science.

[29]  A. Fire,et al.  Distinct roles for RDE-1 and RDE-4 during RNA interference in Caenorhabditis elegans. , 2001, RNA.

[30]  T. Tuschl,et al.  Functional anatomy of siRNAs for mediating efficient RNAi in Drosophila melanogaster embryo lysate , 2001, The EMBO journal.

[31]  A. Caudy,et al.  Role for a bidentate ribonuclease in the initiation step of RNA interference , 2001 .

[32]  P. Sharp,et al.  RNA interference--2001. , 2001, Genes & development.

[33]  K Weber,et al.  Identification of essential genes in cultured mammalian cells using small interfering RNAs. , 2001, Journal of cell science.

[34]  D. Jans,et al.  Gene therapy: optimising DNA delivery to the nucleus. , 2001, Current drug targets.

[35]  T. Tuschl,et al.  Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells , 2001, Nature.

[36]  Brenda L. Bass,et al.  RNA interference: The short answer , 2001, Nature.

[37]  Pirmin Fessler,et al.  References , 1974 .

[38]  J. Birchler,et al.  RNAi related mechanisms affect both transcriptional and posttranscriptional transgene silencing in Drosophila. , 2002, Molecular cell.

[39]  E. Lai Micro RNAs are complementary to 3′ UTR sequence motifs that mediate negative post-transcriptional regulation , 2002, Nature Genetics.

[40]  T. Tuschl,et al.  Identification of Tissue-Specific MicroRNAs from Mouse , 2002, Current Biology.

[41]  David L. Lewis,et al.  Efficient delivery of siRNA for inhibition of gene expression in postnatal mice , 2002, Nature Genetics.

[42]  Stacy L DeRuiter,et al.  RNA interference by expression of short-interfering RNAs and hairpin RNAs in mammalian cells , 2002, Proceedings of the National Academy of Sciences of the United States of America.

[43]  N. Caplen A new approach to the inhibition of gene expression. , 2002, Trends in biotechnology.

[44]  M. Stevenson,et al.  Modulation of HIV-1 replication by RNA interference , 2002, Nature.

[45]  Paul Ahlquist,et al.  RNA-Dependent RNA Polymerases, Viruses, and RNA Silencing , 2002, Science.

[46]  Ali Ehsani,et al.  Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells , 2002, Nature Biotechnology.

[47]  R. Bernards,et al.  Stable suppression of tumorigenicity by virus-mediated RNA interference. , 2002, Cancer cell.

[48]  Henning Urlaub,et al.  Single-Stranded Antisense siRNAs Guide Target RNA Cleavage in RNAi , 2002, Cell.

[49]  G. Hannon RNA interference : RNA , 2002 .

[50]  R. Bernards,et al.  A System for Stable Expression of Short Interfering RNAs in Mammalian Cells , 2002, Science.

[51]  Phillip D Zamore,et al.  RNAi: nature abhors a double-strand. , 2002, Current opinion in genetics & development.

[52]  K. Taira,et al.  U6 promoter–driven siRNAs with four uridine 3′ overhangs efficiently suppress targeted gene expression in mammalian cells , 2002, Nature Biotechnology.

[53]  R. Andino,et al.  Short interfering RNA confers intracellular antiviral immunity in human cells , 2002, Nature.

[54]  W. Forrester,et al.  A DNA vector-based RNAi technology to suppress gene expression in mammalian cells , 2002, Proceedings of the National Academy of Sciences of the United States of America.

[55]  Haibin Xia,et al.  siRNA-mediated gene silencing in vitro and in vivo , 2002, Nature Biotechnology.

[56]  T. Giordano,et al.  RNAi: gene-silencing in therapeutic intervention. , 2002, Drug discovery today.

[57]  A. Fire,et al.  Rescue of polyglutamine-mediated cytotoxicity by double-stranded RNA-mediated RNA interference. , 2002, Human molecular genetics.

[58]  Douglas S. Conklin,et al.  Gene expression: RNA interference in adult mice , 2002, Nature.

[59]  Craig P. Hunter,et al.  Systemic RNAi in C. elegans Requires the Putative Transmembrane Protein SID-1 , 2002, Science.

[60]  K. Kosik,et al.  RNAi functions in cultured mammalian neurons , 2002, Proceedings of the National Academy of Sciences of the United States of America.

[61]  R. Agami RNAi and related mechanisms and their potential use for therapy. , 2002, Current opinion in chemical biology.

[62]  R. Medzhitov,et al.  Retroviral delivery of small interfering RNA into primary cells , 2002, Proceedings of the National Academy of Sciences of the United States of America.

[63]  V. Erdmann,et al.  Comparison of different antisense strategies in mammalian cells using locked nucleic acids, 2'-O-methyl RNA, phosphorothioates and small interfering RNA. , 2003, Nucleic acids research.

[64]  Judy Lieberman,et al.  Interfering with disease: opportunities and roadblocks to harnessing RNA interference , 2003, Trends in Molecular Medicine.

[65]  H. Paulson,et al.  Toward therapy for DYT1 dystonia: Allele‐specific silencing of mutant TorsinA , 2003, Annals of neurology.

[66]  G. Hannon,et al.  RNAi: an ever-growing puzzle. , 2003, Trends in biochemical sciences.

[67]  Michael T. McManus,et al.  A lentivirus-based system to functionally silence genes in primary mammalian cells, stem cells and transgenic mice by RNA interference , 2003, Nature Genetics.

[68]  Mouldy Sioud,et al.  Gene silencing by systemic delivery of synthetic siRNAs in adult mice. , 2003, Journal of molecular biology.

[69]  Petr Svoboda,et al.  RNAi: mammalian oocytes do it without RNA-dependent RNA polymerase. , 2003, RNA.

[70]  Shelley R. Hough,et al.  Why RNAi makes sense , 2003, Nature Biotechnology.

[71]  H. Cerutti,et al.  RNA interference: traveling in the cell and gaining functions? , 2003, Trends in genetics : TIG.

[72]  A. Dillin The specifics of small interfering RNA specificity , 2003, Proceedings of the National Academy of Sciences of the United States of America.

[73]  R. Iggo,et al.  Induction of an interferon response by RNAi vectors in mammalian cells , 2003, Nature Genetics.

[74]  M. Wood,et al.  Therapeutic gene silencing in the nervous system. , 2003, Human molecular genetics.

[75]  M. Wood,et al.  Allele-specific silencing of a pathogenic mutant acetylcholine receptor subunit by RNA interference. , 2003, Human molecular genetics.

[76]  M. Manns,et al.  Caspase 8 small interfering RNA prevents acute liver failure in mice , 2003, Proceedings of the National Academy of Sciences of the United States of America.

[77]  E. Moss Silencing unhealthy alleles naturally. , 2003, Trends in biotechnology.

[78]  Haibin Xia,et al.  Allele-specific silencing of dominant disease genes , 2003, Proceedings of the National Academy of Sciences of the United States of America.

[79]  Peter A. Jones,et al.  Establishment of conditional vectors for hairpin siRNA knockdowns. , 2003, Nucleic acids research.

[80]  Inder M Verma,et al.  A general method for gene knockdown in mice by using lentiviral vectors expressing small interfering RNA , 2003, Proceedings of the National Academy of Sciences of the United States of America.

[81]  V. Kim RNA interference in functional genomics and medicine. , 2003, Journal of Korean medical science.

[82]  J. Church,et al.  siRNA-DIRECTED INHIBITION OF HIV-1 INFECTION , 2003, Pediatrics.

[83]  H. Paulson,et al.  For Personal Use. Only Reproduce with Permission the Lancet Publishing Group. Molecular Medicine for the Brain: Silencing of Disease Genes with Rna Interference , 2022 .

[84]  N. Schütze siRNA technology , 2004, Molecular and Cellular Endocrinology.

[85]  C. Mobbs,et al.  Lobotomy of genes: use of RNA interference in Neuroscience , 2004, Neuroscience.

[86]  K. Tokunaga,et al.  Long‐lasting RNAi activity in mammalian neurons , 2004, FEBS letters.

[87]  R. Plasterk,et al.  RNAi protects the Caenorhabditis elegans germline against transposition. , 2004, Trends in genetics : TIG.

[88]  M. Esteban,et al.  Induction of apoptosis by the dsRNA-dependent protein kinase (PKR): Mechanism of action , 2000, Apoptosis.

[89]  R. Bhatnagar,et al.  siRNA-directed silencing of transgene expressed in cultured insect cells. , 2004, Biochemical and biophysical research communications.

[90]  A. Gartel,et al.  RNA interference as a new strategy against viral hepatitis. , 2004, Virology.

[91]  John J Rossi,et al.  Control of HIV-1 replication by RNA interference. , 2004, Virus research.

[92]  K. Jain RNAi and siRNA in target validation. , 2004, Drug discovery today.

[93]  R. Plasterk,et al.  Dicers at RISC The Mechanism of RNAi , 2004, Cell.