Literature DB >> 33928570

Development of siRNA Therapeutics for the Treatment of Liver Diseases.

Anja Holm1, Marianne Bengtson Løvendorf1, Sakari Kauppinen2.   

Abstract

Small interfering RNA (siRNA)-based therapeutics holds the promise to treat a wide range of human diseases that are currently incurable using conventional therapies. Most siRNA therapeutic efforts to date have focused on the treatment of liver diseases due to major breakthroughs in the development of efficient strategies for delivering siRNA drugs to the liver. Indeed, the development of lipid nanoparticle-formulated and GalNAc-conjugated siRNA therapeutics has resulted in recent FDA approvals of the first siRNA-based drugs, patisiran for the treatment of hereditary transthyretin amyloidosis and givosiran for the treatment of acute hepatic porphyria, respectively. Here, we describe the current strategies for delivering siRNA drugs to the liver and summarize recent advances in clinical development of siRNA therapeutics for the treatment of liver diseases.

Entities:  

Keywords:  ASGPR; Gene silencing; Lipid nanoparticle; Liver; N-Acetylgalactosamine; RNAi; siRNA

Year:  2021        PMID： 33928570     DOI： 10.1007/978-1-0716-1298-9_5

Source DB:  PubMed          Journal:  Methods Mol Biol        ISSN： 1064-3745


  30 in total

1.  Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans.

Authors:  A Fire; S Xu; M K Montgomery; S A Kostas; S E Driver; C C Mello
Journal:  Nature       Date:  1998-02-19       Impact factor: 49.962

Review 2.  The current state and future directions of RNAi-based therapeutics.

Authors:  Ryan L Setten; John J Rossi; Si-Ping Han
Journal:  Nat Rev Drug Discov       Date:  2019-06       Impact factor: 84.694

3.  Single-stranded siRNAs activate RNAi in animals.

Authors:  Walt F Lima; Thazha P Prakash; Heather M Murray; Garth A Kinberger; Wenyu Li; Alfred E Chappell; Cheryl S Li; Susan F Murray; Hans Gaus; Punit P Seth; Eric E Swayze; Stanley T Crooke
Journal:  Cell       Date:  2012-08-31       Impact factor: 41.582

4.  Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis.

Authors:  David Adams; Alejandra Gonzalez-Duarte; William D O'Riordan; Chih-Chao Yang; Mitsuharu Ueda; Arnt V Kristen; Ivailo Tournev; Hartmut H Schmidt; Teresa Coelho; John L Berk; Kon-Ping Lin; Giuseppe Vita; Shahram Attarian; Violaine Planté-Bordeneuve; Michelle M Mezei; Josep M Campistol; Juan Buades; Thomas H Brannagan; Byoung J Kim; Jeeyoung Oh; Yesim Parman; Yoshiki Sekijima; Philip N Hawkins; Scott D Solomon; Michael Polydefkis; Peter J Dyck; Pritesh J Gandhi; Sunita Goyal; Jihong Chen; Andrew L Strahs; Saraswathy V Nochur; Marianne T Sweetser; Pushkal P Garg; Akshay K Vaishnaw; Jared A Gollob; Ole B Suhr
Journal:  N Engl J Med       Date:  2018-07-05       Impact factor: 91.245

5.  Chirality Dependent Potency Enhancement and Structural Impact of Glycol Nucleic Acid Modification on siRNA.

Authors:  Mark K Schlegel; Donald J Foster; Alexander V Kel'in; Ivan Zlatev; Anna Bisbe; Muthusamy Jayaraman; Jeremy G Lackey; Kallanthottathil G Rajeev; Klaus Charissé; Joel Harp; Pradeep S Pallan; Martin A Maier; Martin Egli; Muthiah Manoharan
Journal:  J Am Chem Soc       Date:  2017-06-19       Impact factor: 15.419

6.  5'-(E)-Vinylphosphonate: A Stable Phosphate Mimic Can Improve the RNAi Activity of siRNA-GalNAc Conjugates.

Authors:  Rubina Parmar; Jennifer L S Willoughby; Jingxuan Liu; Donald J Foster; Benjamin Brigham; Christopher S Theile; Klaus Charisse; Akin Akinc; Erin Guidry; Yi Pei; Walter Strapps; Mark Cancilla; Matthew G Stanton; Kallanthottathil G Rajeev; Laura Sepp-Lorenzino; Muthiah Manoharan; Rachel Meyers; Martin A Maier; Vasant Jadhav
Journal:  Chembiochem       Date:  2016-04-28       Impact factor: 3.164

7.  Locked nucleic acid (LNA) mediated improvements in siRNA stability and functionality.

Authors:  Joacim Elmén; Håkan Thonberg; Karl Ljungberg; Miriam Frieden; Majken Westergaard; Yunhe Xu; Britta Wahren; Zicai Liang; Henrik Ørum; Troels Koch; Claes Wahlestedt
Journal:  Nucleic Acids Res       Date:  2005-01-14       Impact factor: 16.971

8.  Analysis of acyclic nucleoside modifications in siRNAs finds sensitivity at position 1 that is restored by 5'-terminal phosphorylation both in vitro and in vivo.

Authors:  Denise M Kenski; Abby J Cooper; Jenny J Li; Aarron T Willingham; Henry J Haringsma; Tracy A Young; Nelly A Kuklin; Jeffrey J Jones; Mark T Cancilla; Daniel R McMasters; Melina Mathur; Alan B Sachs; W Michael Flanagan
Journal:  Nucleic Acids Res       Date:  2009-11-16       Impact factor: 16.971

9.  Effect of asymmetric terminal structures of short RNA duplexes on the RNA interference activity and strand selection.

Authors:  Masayuki Sano; Malgorzata Sierant; Makoto Miyagishi; Mahito Nakanishi; Yasuomi Takagi; Shizuyo Sutou
Journal:  Nucleic Acids Res       Date:  2008-09-09       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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  1 in total

1.  Clinical advances of RNA therapeutics for treatment of neurological and neuromuscular diseases.

Authors:  Anja Holm; Stine N Hansen; Henrik Klitgaard; Sakari Kauppinen
Journal:  RNA Biol       Date:  2021-12-31       Impact factor: 4.766
  1 in total

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