Literature DB >> 29786096

Reversal of siRNA-mediated gene silencing in vivo.

Ivan Zlatev1, Adam Castoreno1, Christopher R Brown1, June Qin1, Scott Waldron1, Mark K Schlegel1, Rohan Degaonkar1, Svetlana Shulga-Morskaya1, Huilei Xu1, Swati Gupta1, Shigeo Matsuda1, Akin Akinc1, Kallanthottathil G Rajeev1, Muthiah Manoharan1, Martin A Maier1, Vasant Jadhav1.   

Abstract

We report rapid, potent reversal of GalNAc-siRNA-mediated RNA interference (RNAi) activity in vivo with short, synthetic, high-affinity oligonucleotides complementary to the siRNA guide strand. We found that 9-mers with five locked nucleic acids (LNAs) have the highest potency across several targets. Our modular, sequence-specific approach, named REVERSIR, may enhance the therapeutic profile of any long-acting GalNAc-siRNA (short interfering RNA) conjugate by enabling control of RNAi pharmacology.

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Year:  2018        PMID： 29786096     DOI： 10.1038/nbt.4136

Source DB:  PubMed          Journal:  Nat Biotechnol        ISSN： 1087-0156            Impact factor:   54.908


  26 in total

1.  Quantitative evaluation of siRNA delivery in vivo.

Authors:  Yi Pei; Paula J Hancock; Hangchun Zhang; René Bartz; Craig Cherrin; Nathalie Innocent; Colin J Pomerantz; Jessica Seitzer; Martin L Koser; Marc T Abrams; Yan Xu; Nelly A Kuklin; Paul A Burke; Alan B Sachs; Laura Sepp-Lorenzino; Stanley F Barnett
Journal:  RNA       Date:  2010-10-12       Impact factor: 4.942

2.  Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate.

Authors:  Tracy S Zimmermann; Verena Karsten; Amy Chan; Joseph Chiesa; Malcolm Boyce; Brian R Bettencourt; Renta Hutabarat; Saraswathy Nochur; Akshay Vaishnaw; Jared Gollob
Journal:  Mol Ther       Date:  2017-01-04       Impact factor: 11.454

3.  STAR: ultrafast universal RNA-seq aligner.

Authors:  Alexander Dobin; Carrie A Davis; Felix Schlesinger; Jorg Drenkow; Chris Zaleski; Sonali Jha; Philippe Batut; Mark Chaisson; Thomas R Gingeras
Journal:  Bioinformatics       Date:  2012-10-25       Impact factor: 6.937

4.  Pharmacological inhibition of a microRNA family in nonhuman primates by a seed-targeting 8-mer antimiR.

Authors:  Veerle Rottiers; Susanna Obad; Andreas Petri; Robert McGarrah; Marie W Lindholm; Joshua C Black; Sumita Sinha; Robin J Goody; Matthew S Lawrence; Andrew S deLemos; Henrik F Hansen; Steve Whittaker; Steve Henry; Rohn Brookes; S Hani Najafi-Shoushtari; Raymond T Chung; Johnathan R Whetstine; Robert E Gerszten; Sakari Kauppinen; Anders M Näär
Journal:  Sci Transl Med       Date:  2013-11-20       Impact factor: 17.956

5.  Silencing of microRNA families by seed-targeting tiny LNAs.

Authors:  Susanna Obad; Camila O dos Santos; Andreas Petri; Markus Heidenblad; Oliver Broom; Cristian Ruse; Cexiong Fu; Morten Lindow; Jan Stenvang; Ellen Marie Straarup; Henrik Frydenlund Hansen; Troels Koch; Darryl Pappin; Gregory J Hannon; Sakari Kauppinen
Journal:  Nat Genet       Date:  2011-03-20       Impact factor: 38.330

6.  Structural basis for microRNA targeting.

Authors:  Nicole T Schirle; Jessica Sheu-Gruttadauria; Ian J MacRae
Journal:  Science       Date:  2014-10-31       Impact factor: 47.728

7.  Treatment of HCV infection by targeting microRNA.

Authors:  Harry L A Janssen; Hendrik W Reesink; Eric J Lawitz; Stefan Zeuzem; Maribel Rodriguez-Torres; Keyur Patel; Adriaan J van der Meer; Amy K Patick; Alice Chen; Yi Zhou; Robert Persson; Barney D King; Sakari Kauppinen; Arthur A Levin; Michael R Hodges
Journal:  N Engl J Med       Date:  2013-03-27       Impact factor: 91.245

8.  Safety and efficacy of RNAi therapy for transthyretin amyloidosis.

Authors:  Teresa Coelho; David Adams; Ana Silva; Pierre Lozeron; Philip N Hawkins; Timothy Mant; Javier Perez; Joseph Chiesa; Steve Warrington; Elizabeth Tranter; Malathy Munisamy; Rick Falzone; Jamie Harrop; Jeffrey Cehelsky; Brian R Bettencourt; Mary Geissler; James S Butler; Alfica Sehgal; Rachel E Meyers; Qingmin Chen; Todd Borland; Renta M Hutabarat; Valerie A Clausen; Rene Alvarez; Kevin Fitzgerald; Christina Gamba-Vitalo; Saraswathy V Nochur; Akshay K Vaishnaw; Dinah W Y Sah; Jared A Gollob; Ole B Suhr
Journal:  N Engl J Med       Date:  2013-08-29       Impact factor: 91.245

9.  Real-time quantification of microRNAs by stem-loop RT-PCR.

Authors:  Caifu Chen; Dana A Ridzon; Adam J Broomer; Zhaohui Zhou; Danny H Lee; Julie T Nguyen; Maura Barbisin; Nan Lan Xu; Vikram R Mahuvakar; Mark R Andersen; Kai Qin Lao; Kenneth J Livak; Karl J Guegler
Journal:  Nucleic Acids Res       Date:  2005-11-27       Impact factor: 16.971

10.  Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity.

Authors:  Maja M Janas; Mark K Schlegel; Carole E Harbison; Vedat O Yilmaz; Yongfeng Jiang; Rubina Parmar; Ivan Zlatev; Adam Castoreno; Huilei Xu; Svetlana Shulga-Morskaya; Kallanthottathil G Rajeev; Muthiah Manoharan; Natalie D Keirstead; Martin A Maier; Vasant Jadhav
Journal:  Nat Commun       Date:  2018-02-19       Impact factor: 14.919
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  12 in total

Review 1.  Delivery of Oligonucleotides to the Liver with GalNAc: From Research to Registered Therapeutic Drug.

Authors:  Alexandre J Debacker; Jon Voutila; Matthew Catley; David Blakey; Nagy Habib
Journal:  Mol Ther       Date:  2020-06-17       Impact factor: 11.454

2.  Alnylam launches era of RNAi drugs.

Authors:  Ken Garber
Journal:  Nat Biotechnol       Date:  2018-09-06       Impact factor: 54.908

3.  RNA interference therapeutics targeting angiotensinogen ameliorate preeclamptic phenotype in rodent models.

Authors:  Nadine Haase; Donald J Foster; Mark W Cunningham; Julia Bercher; Tuyen Nguyen; Svetlana Shulga-Morskaya; Stuart Milstein; Sarfraz Shaikh; Jeff Rollins; Michaela Golic; Florian Herse; Kristin Kräker; Ivo Bendix; Meray Serdar; Hanna Napieczynska; Arnd Heuser; Alexandra Gellhaus; Kristin Thiele; Gerd Wallukat; Dominik N Müller; Babbette LaMarca; Ralf Dechend
Journal:  J Clin Invest       Date:  2020-06-01       Impact factor: 14.808

4.  Augmented lipid-nanoparticle-mediated in vivo genome editing in the lungs and spleen by disrupting Cas9 activity in the liver.

Authors:  Cory D Sago; Melissa P Lokugamage; David Loughrey; Kevin E Lindsay; Robert Hincapie; Brandon R Krupczak; Sujay Kalathoor; Manaka Sato; Elisa Schrader Echeverri; Jordan P Fitzgerald; Zubao Gan; Lena Gamboa; Kalina Paunovska; Carlos A Sanhueza; Marine Z C Hatit; M G Finn; Philip J Santangelo; James E Dahlman
Journal:  Nat Biomed Eng       Date:  2022-02-21       Impact factor: 29.234

Review 5.  The dawn of a new era of targeted lipid-lowering therapies.

Authors:  Lale Tokgözoğlu; Peter Libby
Journal:  Eur Heart J       Date:  2022-09-07       Impact factor: 35.855

Review 6.  The Nonclinical Safety Profile of GalNAc-conjugated RNAi Therapeutics in Subacute Studies.

Authors:  Maja M Janas; Carole E Harbison; Victoria K Perry; Brenda Carito; Jessica E Sutherland; Akshay K Vaishnaw; Natalie D Keirstead; Garvin Warner
Journal:  Toxicol Pathol       Date:  2018-08-23       Impact factor: 1.902

Review 7.  A narrative review of proteolytic targeting chimeras (PROTACs): future perspective for prostate cancer therapy.

Authors:  Xuanrong Chen; Haishan Shen; Yi Shao; Qianwang Ma; Yuanjie Niu; Zhiqun Shang
Journal:  Transl Androl Urol       Date:  2021-02

8.  Docosanoic acid conjugation to siRNA enables functional and safe delivery to skeletal and cardiac muscles.

Authors:  Annabelle Biscans; Jillian Caiazzi; Nicholas McHugh; Vignesh Hariharan; Manish Muhuri; Anastasia Khvorova
Journal:  Mol Ther       Date:  2020-12-19       Impact factor: 11.454

9.  Novel advances in biotransformation and bioactivation research - 2020 year in review.

Authors:  S Cyrus Khojasteh; Upendra A Argikar; James P Driscoll; Carley J S Heck; Lloyd King; Klarissa D Jackson; Wenying Jian; Amit S Kalgutkar; Grover P Miller; Valerie Kramlinger; Ivonne M C M Rietjens; Aaron M Teitelbaum; Kai Wang; Cong Wei
Journal:  Drug Metab Rev       Date:  2021-06-24       Impact factor: 6.984

10.  The chemical structure and phosphorothioate content of hydrophobically modified siRNAs impact extrahepatic distribution and efficacy.

Authors:  Annabelle Biscans; Jillian Caiazzi; Sarah Davis; Nicholas McHugh; Jacquelyn Sousa; Anastasia Khvorova
Journal:  Nucleic Acids Res       Date:  2020-08-20       Impact factor: 16.971
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