Literature DB >> 34931905

Challenges and Opportunities for Nucleic Acid Therapeutics.

David R Corey1, Masad J Damha2, Muthiah Manoharan3.   

Abstract

After decades overcoming difficult problems, antisense oligonucleotide (ASO), duplex RNA (siRNA), and messenger RNA (mRNA) nucleic acid therapeutic strategies are finally demonstrating clinical benefits. This success presents new challenges. What goals remain for basic research? Will there be an explosion of clinical applications that benefit many patients with different diseases, or will success be restricted to diseases that are ideal for the application of current technologies? The aim of this perspective is to describe a selection of the major goals for the next decade.

Entities:  

Keywords:  antisense; siRNA; therapeutic

Mesh：

Substances：

Year:  2021        PMID： 34931905      PMCID： PMC8817707          DOI： 10.1089/nat.2021.0085

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


  37 in total

1.  'N of 1' therapies need a better model.

Authors:  Annemieke Aartsma-Rus
Journal:  Nat Med       Date:  2021-05-24       Impact factor: 53.440

2.  Messenger RNA therapy as an option for treating metabolic disorders.

Authors:  Randy J Chandler
Journal:  Proc Natl Acad Sci U S A       Date:  2019-09-19       Impact factor: 11.205

3.  Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide.

Authors:  P C Zamecnik; M L Stephenson
Journal:  Proc Natl Acad Sci U S A       Date:  1978-01       Impact factor: 11.205

4.  Multivalent N-acetylgalactosamine-conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencing.

Authors:  Jayaprakash K Nair; Jennifer L S Willoughby; Amy Chan; Klaus Charisse; Md Rowshon Alam; Qianfan Wang; Menno Hoekstra; Pachamuthu Kandasamy; Alexander V Kel'in; Stuart Milstein; Nate Taneja; Jonathan O'Shea; Sarfraz Shaikh; Ligang Zhang; Ronald J van der Sluis; Michael E Jung; Akin Akinc; Renta Hutabarat; Satya Kuchimanchi; Kevin Fitzgerald; Tracy Zimmermann; Theo J C van Berkel; Martin A Maier; Kallanthottathil G Rajeev; Muthiah Manoharan
Journal:  J Am Chem Soc       Date:  2014-12-01       Impact factor: 15.419

5.  Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.

Authors:  Jinkuk Kim; Chunguang Hu; Christelle Moufawad El Achkar; Lauren E Black; Julie Douville; Austin Larson; Mary K Pendergast; Sara F Goldkind; Eunjung A Lee; Ashley Kuniholm; Aubrie Soucy; Jai Vaze; Nandkishore R Belur; Kristina Fredriksen; Iva Stojkovska; Alla Tsytsykova; Myriam Armant; Renata L DiDonato; Jaejoon Choi; Laura Cornelissen; Luis M Pereira; Erika F Augustine; Casie A Genetti; Kira Dies; Brenda Barton; Lucinda Williams; Benjamin D Goodlett; Bobbie L Riley; Amy Pasternak; Emily R Berry; Kelly A Pflock; Stephen Chu; Chantal Reed; Kimberly Tyndall; Pankaj B Agrawal; Alan H Beggs; P Ellen Grant; David K Urion; Richard O Snyder; Susan E Waisbren; Annapurna Poduri; Peter J Park; Al Patterson; Alessandra Biffi; Joseph R Mazzulli; Olaf Bodamer; Charles B Berde; Timothy W Yu
Journal:  N Engl J Med       Date:  2019-10-09       Impact factor: 91.245

6.  Long-term mipomersen treatment is associated with a reduction in cardiovascular events in patients with familial hypercholesterolemia.

Authors:  P Barton Duell; Raul D Santos; Bridget-Anne Kirwan; Joseph L Witztum; Sotirios Tsimikas; John J P Kastelein
Journal:  J Clin Lipidol       Date:  2016-05-09       Impact factor: 4.766

Review 7.  Is very low LDL-C harmful?

Authors:  Charles Faselis; Konstantinos Imprialos; Haris Grassos; Andreas Pittaras; Manolis Kallistratos; Athanasios Manolis
Journal:  Curr Pharm Des       Date:  2018       Impact factor: 3.116

Review 8.  Spinal Muscular Atrophy.

Authors:  Stefan Nicolau; Megan A Waldrop; Anne M Connolly; Jerry R Mendell
Journal:  Semin Pediatr Neurol       Date:  2021-02-11       Impact factor: 1.636

9.  Argonaute binding within 3'-untranslated regions poorly predicts gene repression.

Authors:  Yongjun Chu; Audrius Kilikevicius; Jing Liu; Krystal C Johnson; Shinnichi Yokota; David R Corey
Journal:  Nucleic Acids Res       Date:  2020-07-27       Impact factor: 16.971

10.  Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice.

Authors:  Thazha P Prakash; Mark J Graham; Jinghua Yu; Rick Carty; Audrey Low; Alfred Chappell; Karsten Schmidt; Chenguang Zhao; Mariam Aghajan; Heather F Murray; Stan Riney; Sheri L Booten; Susan F Murray; Hans Gaus; Jeff Crosby; Walt F Lima; Shuling Guo; Brett P Monia; Eric E Swayze; Punit P Seth
Journal:  Nucleic Acids Res       Date:  2014-07-03       Impact factor: 16.971
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  2 in total

1.  Aminooxy Click Chemistry as a Tool for Bis-homo and Bis-hetero Ligand Conjugation to Nucleic Acids.

Authors:  Dhrubajyoti Datta; Shohei Mori; Mimouna Madaoui; Kelly Wassarman; Ivan Zlatev; Muthiah Manoharan
Journal:  Org Lett       Date:  2022-06-17       Impact factor: 6.072

2.  DNA/RNA heteroduplex oligonucleotides: An unanticipated twist in the delivery of ASOs.

Authors:  Ryan L Setten; Steven F Dowdy
Journal:  Mol Ther Nucleic Acids       Date:  2022-07-01       Impact factor: 10.183
  2 in total

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