Literature DB >> 28374018

Antisense Oligonucleotides: Treatment Strategies and Cellular Internalization.

Colton M Miller1, Edward N Harris1.   

Abstract

The clinical applicaton of antisense oligonucleotides (ASOs) is becoming more of a reality as several drugs have been approved for the treatment of human disorders and many others are in various phases in development and clinical trials. ASOs are short DNA/RNA oligos which are heavily modified to increase their stability in biological fluids and retain the properties of creating RNA-RNA and DNA-RNA duplexes that knock-down or correct genetic expression. This review outlines several strategies that ASOs utilize for the treatment of various congenital diseases and syndromes that develop with aging. In addition, we discuss some of the mechanisms for specific non-targeted ASO internalization within cells.

Entities:  

Keywords:  Stabilin; antisense oligonucleotide; clearance; endocytosis; phosphorothioate; splicing

Year:  2016        PMID： 28374018      PMCID： PMC5376066          DOI： 10.14800/rd.1393

Source DB:  PubMed          Journal:  RNA Dis        ISSN： 2375-2467


  56 in total

1.  Expression, processing, and glycosaminoglycan binding activity of the recombinant human 315-kDa hyaluronic acid receptor for endocytosis (HARE).

Authors:  Edward N Harris; Svetlana V Kyosseva; Janet A Weigel; Paul H Weigel
Journal:  J Biol Chem       Date:  2006-12-04       Impact factor: 5.157

2.  Binding, uptake, and intracellular trafficking of phosphorothioate-modified oligodeoxynucleotides.

Authors:  C Beltinger; H U Saragovi; R M Smith; L LeSauteur; N Shah; L DeDionisio; L Christensen; A Raible; L Jarett; A M Gewirtz
Journal:  J Clin Invest       Date:  1995-04       Impact factor: 14.808

3.  Nonionic nucleic acid analogues. Synthesis and characterization of dideoxyribonucleoside methylphosphonates.

Authors:  P S Miller; J Yano; E Yano; C Carroll; K Jayaraman; P O Ts'o
Journal:  Biochemistry       Date:  1979-11-13       Impact factor: 3.162

4.  Exon skipping quantification by quantitative reverse-transcription polymerase chain reaction in Duchenne muscular dystrophy patients treated with the antisense oligomer eteplirsen.

Authors:  Karen Anthony; Lucy Feng; Virginia Arechavala-Gomeza; Michela Guglieri; Volker Straub; Katherine Bushby; Sebahattin Cirak; Jennifer Morgan; Francesco Muntoni
Journal:  Hum Gene Ther Methods       Date:  2012-10       Impact factor: 2.396

5.  Antisense therapy targeting apolipoprotein(a): a randomised, double-blind, placebo-controlled phase 1 study.

Authors:  Sotirios Tsimikas; Nicholas J Viney; Steven G Hughes; Walter Singleton; Mark J Graham; Brenda F Baker; Jennifer L Burkey; Qingqing Yang; Santica M Marcovina; Richard S Geary; Rosanne M Crooke; Joseph L Witztum
Journal:  Lancet       Date:  2015-07-22       Impact factor: 79.321

6.  Phosphorothioate oligodeoxynucleotides bind to basic fibroblast growth factor, inhibit its binding to cell surface receptors, and remove it from low affinity binding sites on extracellular matrix.

Authors:  M A Guvakova; L A Yakubov; I Vlodavsky; J L Tonkinson; C A Stein
Journal:  J Biol Chem       Date:  1995-02-10       Impact factor: 5.157

7.  Pharmacokinetic and Pharmacodynamic Investigations of ION-353382, a Model Antisense Oligonucleotide: Using Alpha-2-Macroglobulin and Murinoglobulin Double-Knockout Mice.

Authors:  Colby S Shemesh; Rosie Z Yu; Hans J Gaus; Punit P Seth; Eric E Swayze; Frank C Bennett; Richard S Geary; Scott P Henry; Yanfeng Wang
Journal:  Nucleic Acid Ther       Date:  2016-03-31       Impact factor: 5.486

8.  Inhibition of high affinity basic fibroblast growth factor binding by oligonucleotides.

Authors:  S M Fennewald; R F Rando
Journal:  J Biol Chem       Date:  1995-09-15       Impact factor: 5.157

9.  The DcpS inhibitor RG3039 improves motor function in SMA mice.

Authors:  James P Van Meerbeke; Rebecca M Gibbs; Heather L Plasterer; Wenyan Miao; Zhihua Feng; Ming-Yi Lin; Agnieszka A Rucki; Claribel D Wee; Bing Xia; Shefali Sharma; Vincent Jacques; Darrick K Li; Livio Pellizzoni; James R Rusche; Chien-Ping Ko; Charlotte J Sumner
Journal:  Hum Mol Genet       Date:  2013-05-31       Impact factor: 6.150

10.  Synthetic oligonucleotides recruit ILF2/3 to RNA transcripts to modulate splicing.

Authors:  Frank Rigo; Yimin Hua; Seung J Chun; Thazha P Prakash; Adrian R Krainer; C Frank Bennett
Journal:  Nat Chem Biol       Date:  2012-04-15       Impact factor: 15.040
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  14 in total

Review 1.  Receptor-Mediated Uptake of Phosphorothioate Antisense Oligonucleotides in Different Cell Types of the Liver.

Authors:  Colton M Miller; Michael Tanowitz; Aaron J Donner; Thazha P Prakash; Eric E Swayze; Edward N Harris; Punit P Seth
Journal:  Nucleic Acid Ther       Date:  2018-02-09       Impact factor: 5.486

Review 2.  Neurodegenerative diseases associated with non-coding CGG tandem repeat expansions.

Authors:  Zhi-Dong Zhou; Joseph Jankovic; Tetsuo Ashizawa; Eng-King Tan
Journal:  Nat Rev Neurol       Date:  2022-01-12       Impact factor: 44.711

3.  Endosomal Escape of Antisense Oligonucleotides Internalized by Stabilin Receptors Is Regulated by Rab5C and EEA1 During Endosomal Maturation.

Authors:  Colton M Miller; W Brad Wan; Punit P Seth; Edward N Harris
Journal:  Nucleic Acid Ther       Date:  2018-02-13       Impact factor: 5.486

4.  Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice.

Authors:  Arnaud F Klein; Miguel A Varela; Ludovic Arandel; Ashling Holland; Naira Naouar; Andrey Arzumanov; David Seoane; Lucile Revillod; Guillaume Bassez; Arnaud Ferry; Dominic Jauvin; Genevieve Gourdon; Jack Puymirat; Michael J Gait; Denis Furling; Matthew Ja Wood
Journal:  J Clin Invest       Date:  2019-11-01       Impact factor: 14.808

5.  Potent antitumor effects of cell-penetrating peptides targeting STAT3 axis.

Authors:  Maryam Aftabizadeh; Yi-Jia Li; Qianqian Zhao; Chunyan Zhang; Nigus Ambaye; Jieun Song; Toshikage Nagao; Christoph Lahtz; Marwan Fakih; David K Ann; Hua Yu; Andreas Herrmann
Journal:  JCI Insight       Date:  2021-01-25

6.  Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts.

Authors:  Joshua Lee; Yusuke Echigoya; William Duddy; Takashi Saito; Yoshitsugu Aoki; Shin'ichi Takeda; Toshifumi Yokota
Journal:  PLoS One       Date:  2018-05-17       Impact factor: 3.240

Review 7.  Immunomodulation in Cystic Fibrosis: Why and How?

Authors:  Vincent D Giacalone; Brian S Dobosh; Amit Gaggar; Rabindra Tirouvanziam; Camilla Margaroli
Journal:  Int J Mol Sci       Date:  2020-05-08       Impact factor: 5.923

Review 8.  Ligand Binding and Signaling of HARE/Stabilin-2.

Authors:  Edward N Harris; Fatima Cabral
Journal:  Biomolecules       Date:  2019-07-11

Review 9.  Long Non-Coding RNA HOTAIR in Breast Cancer Therapy.

Authors:  Monica Cantile; Maurizio Di Bonito; Margherita Cerrone; Francesca Collina; Michelino De Laurentiis; Gerardo Botti
Journal:  Cancers (Basel)       Date:  2020-05-09       Impact factor: 6.639

10.  The Potential of Telomeric G-quadruplexes Containing Modified Oligoguanosine Overhangs in Activation of Bacterial Phagocytosis and Leukotriene Synthesis in Human Neutrophils.

Authors:  Ekaterina A Golenkina; Galina M Viryasova; Nina G Dolinnaya; Valeria A Bannikova; Tatjana V Gaponova; Yulia M Romanova; Galina F Sud'ina
Journal:  Biomolecules       Date:  2020-02-06
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