Literature DB >> 22703279

RNA interference trigger variants: getting the most out of RNA for RNA interference-based therapeutics.

Nicholas M Snead1, John J Rossi.   

Abstract

The manifestation of RNA interference (RNAi)-based therapeutics lies in safe and successful delivery of small interfering RNAs (siRNAs), the molecular entity that triggers and guides sequence-specific degradation of target mRNAs. Optimizing the chemistry and structure of siRNAs to achieve maximum efficacy is an important parameter in the development of siRNA therapeutics. The RNAi protein machinery can tolerate a variety of non-canonical modifications made to siRNAs, each of which imparts advantageous properties. Here, we review these modifications to siRNAs in pre-clinical and clinical studies.

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Year:  2012        PMID： 22703279      PMCID： PMC3423874          DOI： 10.1089/nat.2012.0361

Source DB:  PubMed          Journal:  Nucleic Acid Ther        ISSN： 2159-3337            Impact factor:   5.486


  77 in total

1.  Longer 19-base pair short interfering RNA duplexes rather than shorter duplexes trigger RNA interference.

Authors:  Malgorzata Sierant; Julia Kazmierczak-Baranska; Alina Paduszynska; Milena Sobczak; Aleksandra Pietkiewicz; Barbara Nawrot
Journal:  Oligonucleotides       Date:  2010-08

Review 2.  Strategies for silencing human disease using RNA interference.

Authors:  Daniel H Kim; John J Rossi
Journal:  Nat Rev Genet       Date:  2007-03       Impact factor: 53.242

3.  Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs.

Authors:  Jürgen Soutschek; Akin Akinc; Birgit Bramlage; Klaus Charisse; Rainer Constien; Mary Donoghue; Sayda Elbashir; Anke Geick; Philipp Hadwiger; Jens Harborth; Matthias John; Venkitasamy Kesavan; Gary Lavine; Rajendra K Pandey; Timothy Racie; Kallanthottathil G Rajeev; Ingo Röhl; Ivanka Toudjarska; Gang Wang; Silvio Wuschko; David Bumcrot; Victor Koteliansky; Stefan Limmer; Muthiah Manoharan; Hans-Peter Vornlocher
Journal:  Nature       Date:  2004-11-11       Impact factor: 49.962

4.  Inhibition of CD44 gene expression in human skin models, using self-delivery short interfering RNA administered by dissolvable microneedle arrays.

Authors:  Maria Fernanda Lara; Emilio González-González; Tycho J Speaker; Robyn P Hickerson; Devin Leake; Leonard M Milstone; Christopher H Contag; Roger L Kaspar
Journal:  Hum Gene Ther       Date:  2012-06-05       Impact factor: 5.695

5.  Protection against TNFα-dependent liver toxicity by intraperitoneal liposome delivered DsiRNA targeting TNFα in vivo.

Authors:  Patric Lundberg; Hui-Jung Yang; Seung-Jae Jung; Mark A Behlke; Scott D Rose; Edouard M Cantin
Journal:  J Control Release       Date:  2011-11-07       Impact factor: 9.776

6.  Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles.

Authors:  Mark E Davis; Jonathan E Zuckerman; Chung Hang J Choi; David Seligson; Anthony Tolcher; Christopher A Alabi; Yun Yen; Jeremy D Heidel; Antoni Ribas
Journal:  Nature       Date:  2010-03-21       Impact factor: 49.962

7.  Construction of folate-conjugated pRNA of bacteriophage phi29 DNA packaging motor for delivery of chimeric siRNA to nasopharyngeal carcinoma cells.

Authors:  S Guo; F Huang; P Guo
Journal:  Gene Ther       Date:  2006-05       Impact factor: 5.250

8.  Single-nucleotide-specific siRNA targeting in a dominant-negative skin model.

Authors:  Robyn P Hickerson; Frances J D Smith; Robert E Reeves; Christopher H Contag; Devin Leake; Sancy A Leachman; Leonard M Milstone; W H Irwin McLean; Roger L Kaspar
Journal:  J Invest Dermatol       Date:  2007-10-11       Impact factor: 8.551

9.  A large-scale chemical modification screen identifies design rules to generate siRNAs with high activity, high stability and low toxicity.

Authors:  Jesper B Bramsen; Maria B Laursen; Anne F Nielsen; Thomas B Hansen; Claus Bus; Niels Langkjaer; B Ravindra Babu; Torben Højland; Mikhail Abramov; Arthur Van Aerschot; Dalibor Odadzic; Romualdas Smicius; Jens Haas; Cordula Andree; Jharna Barman; Malgorzata Wenska; Puneet Srivastava; Chuanzheng Zhou; Dmytro Honcharenko; Simone Hess; Elke Müller; Georgii V Bobkov; Sergey N Mikhailov; Eugenio Fava; Thomas F Meyer; Jyoti Chattopadhyaya; Marino Zerial; Joachim W Engels; Piet Herdewijn; Jesper Wengel; Jørgen Kjems
Journal:  Nucleic Acids Res       Date:  2009-03-12       Impact factor: 16.971

Review 10.  Interfering with disease: a progress report on siRNA-based therapeutics.

Authors:  Antonin de Fougerolles; Hans-Peter Vornlocher; John Maraganore; Judy Lieberman
Journal:  Nat Rev Drug Discov       Date:  2007-06       Impact factor: 84.694
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  12 in total

Review 1.  Technologies for controlled, local delivery of siRNA.

Authors:  Samantha M Sarett; Christopher E Nelson; Craig L Duvall
Journal:  J Control Release       Date:  2015-11-28       Impact factor: 9.776

Review 2.  Technologies for investigating the physiological barriers to efficient lipid nanoparticle-siRNA delivery.

Authors:  Bin Shi; Marc Abrams
Journal:  J Histochem Cytochem       Date:  2013-03-14       Impact factor: 2.479

3.  TLR9-Targeted SiRNA Delivery In Vivo.

Authors:  Dewan Md Sakib Hossain; Dayson Moreira; Qifang Zhang; Sergey Nechaev; Piotr Swiderski; Marcin Kortylewski
Journal:  Methods Mol Biol       Date:  2016

4.  Lipid nanoparticle delivery systems for siRNA-based therapeutics.

Authors:  C Wan; T M Allen; P R Cullis
Journal:  Drug Deliv Transl Res       Date:  2014-02       Impact factor: 4.617

5.  Transcriptome-wide-scale-predicted dsRNAs potentially involved in RNA homoeostasis are remarkably excluded from genes with no/very low expression in all developmental stages.

Authors:  Claude Pasquier; Sandra Agnel; Alain Robichon
Journal:  RNA Biol       Date:  2020-02-04       Impact factor: 4.652

6.  Effective inhibition of HIV-1 production by short hairpin RNAs and small interfering RNAs targeting a highly conserved site in HIV-1 Gag RNA is optimized by evaluating alternative length formats.

Authors:  Robert J Scarborough; Kelsey L Adams; Aïcha Daher; Anne Gatignol
Journal:  Antimicrob Agents Chemother       Date:  2015-06-15       Impact factor: 5.191

7.  Molecular basis for improved gene silencing by Dicer substrate interfering RNA compared with other siRNA variants.

Authors:  Nicholas M Snead; Xiwei Wu; Arthur Li; Qi Cui; Kumi Sakurai; John C Burnett; John J Rossi
Journal:  Nucleic Acids Res       Date:  2013-04-24       Impact factor: 16.971

8.  Optimal choice of functional and off-target effect-reduced siRNAs for RNAi therapeutics.

Authors:  Kumiko Ui-Tei
Journal:  Front Genet       Date:  2013-06-11       Impact factor: 4.599

9.  Inhibition of Sox2 Expression in the Adult Neural Stem Cell Niche In Vivo by Monocationic-based siRNA Delivery.

Authors:  Sylvie Remaud; Silvia Alejandra López-Juárez; Anne-Laure Bolcato-Bellemin; Patrick Neuberg; Fabrice Stock; Marie-Elise Bonnet; Rym Ghaddab; Marie Stéphanie Clerget-Froidevaux; Jacqueline Pierre-Simons; Patrick Erbacher; Barbara A Demeneix; Ghislaine Morvan-Dubois
Journal:  Mol Ther Nucleic Acids       Date:  2013-04-23       Impact factor: 10.183

10.  Design of siRNA Therapeutics from the Molecular Scale.

Authors:  Phillip Angart; Daniel Vocelle; Christina Chan; S Patrick Walton
Journal:  Pharmaceuticals (Basel)       Date:  2013
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