Literature DB >> 26423833

Antisense oligonucleotide drugs for Duchenne muscular dystrophy: how far have we come and what does the future hold?

Ashley Guncay1, Toshifumi Yokota1,2.   

Abstract

Entities:  

Keywords:  2′-O methyl phosphorothioate antisense oligonucleotides; Duchenne muscular dystrophy; antisense therapy; drisapersen; eteplirsen; exon skipping; phosphorodiamidate morpholino oligomers; tricyclo-DNA oligomers

Mesh：

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Year:  2015        PMID： 26423833     DOI： 10.4155/fmc.15.116

Source DB:  PubMed          Journal:  Future Med Chem        ISSN： 1756-8919            Impact factor:   3.808


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  7 in total

Review 1.  Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy.

Authors:  Kasia Dzierlega; Toshifumi Yokota
Journal:  Gene Ther       Date:  2020-06-01       Impact factor: 5.250

Review 2.  Immortalized Muscle Cell Model to Test the Exon Skipping Efficacy for Duchenne Muscular Dystrophy.

Authors:  Quynh Nguyen; Toshifumi Yokota
Journal:  J Pers Med       Date:  2017-10-16

Review 3.  Current Translational Research and Murine Models For Duchenne Muscular Dystrophy.

Authors:  Merryl Rodrigues; Yusuke Echigoya; So-Ichiro Fukada; Toshifumi Yokota
Journal:  J Neuromuscul Dis       Date:  2016-03-03

Review 4.  Skipping Multiple Exons to Treat DMD-Promises and Challenges.

Authors:  Tejal Aslesh; Rika Maruyama; Toshifumi Yokota
Journal:  Biomedicines       Date:  2018-01-02

5.  A novel mouse model of PMS2 founder mutation that causes mismatch repair defect due to aberrant splicing.

Authors:  Kajal Biswas; Martin Couillard; Luca Cavallone; Sandra Burkett; Stacey Stauffer; Betty K Martin; Eileen Southon; Susan Reid; Teri M Plona; Ryan N Baugher; Stephanie D Mellott; Kristen M Pike; Mary E Albaugh; Chelsea Maedler-Kron; Nancy Hamel; Lino Tessarollo; Victoria Marcus; William D Foulkes; Shyam K Sharan
Journal:  Cell Death Dis       Date:  2021-09-06       Impact factor: 8.469

6.  Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy.

Authors:  Bailey Miskew Nichols; Yoshitsugu Aoki; Mutsuki Kuraoka; Joshua J A Lee; Shin'ichi Takeda; Toshifumi Yokota
Journal:  J Vis Exp       Date:  2016-05-24       Impact factor: 1.355

Review 7.  Eteplirsen in the treatment of Duchenne muscular dystrophy.

Authors:  Kenji Rowel Q Lim; Rika Maruyama; Toshifumi Yokota
Journal:  Drug Des Devel Ther       Date:  2017-02-28       Impact factor: 4.162
  7 in total

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