Literature DB >> 24506781

Exon-skipping antisense oligonucleotides to correct missplicing in neurogenetic diseases.

Kavitha Siva1, Giuseppina Covello, Michela A Denti.   

Abstract

Alternative splicing is an important regulator of the transcriptome. However, mutations may cause alteration of splicing patterns, which in turn leads to disease. During the past 10 years, exon skipping has been looked upon as a powerful tool for correction of missplicing in disease and progress has been made towards clinical trials. In this review, we discuss the use of antisense oligonucleotides to correct splicing defects through exon skipping, with a special focus on diseases affecting the nervous system, and the latest stage achieved in its progress.

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Year:  2014        PMID： 24506781      PMCID： PMC3922311          DOI： 10.1089/nat.2013.0461

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


  132 in total

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Journal:  Adv Drug Deliv Rev       Date:  2011-11-07       Impact factor: 15.470

2.  PNA hybridizes to complementary oligonucleotides obeying the Watson-Crick hydrogen-bonding rules.

Authors:  M Egholm; O Buchardt; L Christensen; C Behrens; S M Freier; D A Driver; R H Berg; S K Kim; B Norden; P E Nielsen
Journal:  Nature       Date:  1993-10-07       Impact factor: 49.962

3.  Up-regulation of luciferase gene expression with antisense oligonucleotides: implications and applications in functional assay development.

Authors:  S H Kang; M J Cho; R Kole
Journal:  Biochemistry       Date:  1998-05-05       Impact factor: 3.162

4.  A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse.

Authors:  Paul N Porensky; Chalermchai Mitrpant; Vicki L McGovern; Adam K Bevan; Kevin D Foust; Brain K Kaspar; Stephen D Wilton; Arthur H M Burghes
Journal:  Hum Mol Genet       Date:  2011-12-20       Impact factor: 6.150

5.  In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping.

Authors:  Hans A Heemskerk; Christa L de Winter; Sjef J de Kimpe; Petra van Kuik-Romeijn; Niki Heuvelmans; Gerard J Platenburg; Gert-Jan B van Ommen; Judith C T van Deutekom; Annemieke Aartsma-Rus
Journal:  J Gene Med       Date:  2009-03       Impact factor: 4.565

6.  Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice.

Authors:  Haifang Yin; Qilong Lu; Matthew Wood
Journal:  Mol Ther       Date:  2007-10-30       Impact factor: 11.454

7.  Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice.

Authors:  Natee Jearawiriyapaisarn; Hong M Moulton; Brian Buckley; Jennifer Roberts; Peter Sazani; Suthat Fucharoen; Patrick L Iversen; Ryszard Kole
Journal:  Mol Ther       Date:  2008-06-10       Impact factor: 11.454

8.  Nature and mRNA effect of 282 different NF1 point mutations: focus on splicing alterations.

Authors:  Eva Pros; Carolina Gómez; Thamar Martín; Pere Fábregas; Eduard Serra; Conxi Lázaro
Journal:  Hum Mutat       Date:  2008-09       Impact factor: 4.878

Review 9.  Splicing therapy for neuromuscular disease.

Authors:  Andrew G L Douglas; Matthew J A Wood
Journal:  Mol Cell Neurosci       Date:  2013-04-28       Impact factor: 4.314

10.  Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function.

Authors:  HaiFang Yin; Hong M Moulton; Yiqi Seow; Corinne Boyd; Jordan Boutilier; Patrick Iverson; Matthew J A Wood
Journal:  Hum Mol Genet       Date:  2008-09-10       Impact factor: 6.150
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  26 in total

1.  Analysis of the functional consequences of targeted exon deletion in COL7A1 reveals prospects for dystrophic epidermolysis bullosa therapy.

Authors:  Olivier Bornert; Tobias Kühl; Jeroen Bremer; Peter C van den Akker; Anna Mg Pasmooij; Alexander Nyström
Journal:  Mol Ther       Date:  2016-05-09       Impact factor: 11.454

2.  Exon skipping of FcεRIβ eliminates expression of the high-affinity IgE receptor in mast cells with therapeutic potential for allergy.

Authors:  Glenn Cruse; Yuzhi Yin; Tomoki Fukuyama; Avanti Desai; Greer K Arthur; Wolfgang Bäumer; Michael A Beaven; Dean D Metcalfe
Journal:  Proc Natl Acad Sci U S A       Date:  2016-11-21       Impact factor: 11.205

Review 3.  The role of splicing factors in deregulation of alternative splicing during oncogenesis and tumor progression.

Authors:  Asaf Shilo; Zahava Siegfried; Rotem Karni
Journal:  Mol Cell Oncol       Date:  2014-12-01

Review 4.  Changing the Paradigm - Treating the Basic Defect in Cystic Fibrosis.

Authors:  Lokesh Guglani
Journal:  Indian J Pediatr       Date:  2015-06-17       Impact factor: 1.967

Review 5.  Cerebral Organoids and Antisense Oligonucleotide Therapeutics: Challenges and Opportunities.

Authors:  Jenny Lange; Haiyan Zhou; Amy McTague
Journal:  Front Mol Neurosci       Date:  2022-06-27       Impact factor: 6.261

Review 6.  Lysosomal storage diseases.

Authors:  Frances M Platt; Alessandra d'Azzo; Beverly L Davidson; Elizabeth F Neufeld; Cynthia J Tifft
Journal:  Nat Rev Dis Primers       Date:  2018-10-01       Impact factor: 52.329

7.  Perfluoroaryl Bicyclic Cell-Penetrating Peptides for Delivery of Antisense Oligonucleotides.

Authors:  Justin M Wolfe; Colin M Fadzen; Rebecca L Holden; Monica Yao; Gunnar J Hanson; Bradley L Pentelute
Journal:  Angew Chem Int Ed Engl       Date:  2018-03-14       Impact factor: 15.336

8.  Development and Application of an Ultrasensitive Hybridization-Based ELISA Method for the Determination of Peptide-Conjugated Phosphorodiamidate Morpholino Oligonucleotides.

Authors:  Umar Burki; Jonathan Keane; Alison Blain; Liz O'Donovan; Michael John Gait; Steven H Laval; Volker Straub
Journal:  Nucleic Acid Ther       Date:  2015-07-15       Impact factor: 5.486

9.  eSkip-Finder: a machine learning-based web application and database to identify the optimal sequences of antisense oligonucleotides for exon skipping.

Authors:  Shuntaro Chiba; Kenji Rowel Q Lim; Narin Sheri; Saeed Anwar; Esra Erkut; Md Nur Ahad Shah; Tejal Aslesh; Stanley Woo; Omar Sheikh; Rika Maruyama; Hiroaki Takano; Katsuhiko Kunitake; William Duddy; Yasushi Okuno; Yoshitsugu Aoki; Toshifumi Yokota
Journal:  Nucleic Acids Res       Date:  2021-07-02       Impact factor: 16.971

Review 10.  An alternative splicing hypothesis for neuropathology of schizophrenia: evidence from studies on historical candidate genes and multi-omics data.

Authors:  Chu-Yi Zhang; Xiao Xiao; Zhuohua Zhang; Zhonghua Hu; Ming Li
Journal:  Mol Psychiatry       Date:  2021-03-08       Impact factor: 15.992
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