Literature DB >> 28893959

Mutation-Based Therapy for Duchenne Muscular Dystrophy: Antisense Treatment Arrives in the Clinic.

Elizabeth M McNally1, Eugene J Wyatt2.   

Abstract

Entities:  

Keywords:  exons; genetics

Mesh：

Substances：

Year:  2017        PMID： 28893959      PMCID： PMC5657589          DOI： 10.1161/CIRCULATIONAHA.117.028382

Source DB:  PubMed          Journal:  Circulation        ISSN： 0009-7322            Impact factor:   29.690


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  5 in total

1.  DMD genotypes and loss of ambulation in the CINRG Duchenne Natural History Study.

Authors:  Luca Bello; Lauren P Morgenroth; Heather Gordish-Dressman; Eric P Hoffman; Craig M McDonald; Sebahattin Cirak
Journal:  Neurology       Date:  2016-06-24       Impact factor: 9.910

2.  The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations.

Authors:  Catherine L Bladen; David Salgado; Soledad Monges; Maria E Foncuberta; Kyriaki Kekou; Konstantina Kosma; Hugh Dawkins; Leanne Lamont; Anna J Roy; Teodora Chamova; Velina Guergueltcheva; Sophelia Chan; Lawrence Korngut; Craig Campbell; Yi Dai; Jen Wang; Nina Barišić; Petr Brabec; Jaana Lahdetie; Maggie C Walter; Olivia Schreiber-Katz; Veronika Karcagi; Marta Garami; Venkatarman Viswanathan; Farhad Bayat; Filippo Buccella; En Kimura; Zaïda Koeks; Janneke C van den Bergen; Miriam Rodrigues; Richard Roxburgh; Anna Lusakowska; Anna Kostera-Pruszczyk; Janusz Zimowski; Rosário Santos; Elena Neagu; Svetlana Artemieva; Vedrana Milic Rasic; Dina Vojinovic; Manuel Posada; Clemens Bloetzer; Pierre-Yves Jeannet; Franziska Joncourt; Jordi Díaz-Manera; Eduard Gallardo; A Ayşe Karaduman; Haluk Topaloğlu; Rasha El Sherif; Angela Stringer; Andriy V Shatillo; Ann S Martin; Holly L Peay; Matthew I Bellgard; Jan Kirschner; Kevin M Flanigan; Volker Straub; Kate Bushby; Jan Verschuuren; Annemieke Aartsma-Rus; Christophe Béroud; Hanns Lochmüller
Journal:  Hum Mutat       Date:  2015-03-17       Impact factor: 4.878

3.  Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study.

Authors:  Nathalie M Goemans; Már Tulinius; Marleen van den Hauwe; Anna-Karin Kroksmark; Gunnar Buyse; Rosamund J Wilson; Judith C van Deutekom; Sjef J de Kimpe; Afrodite Lourbakos; Giles Campion
Journal:  PLoS One       Date:  2016-09-02       Impact factor: 3.240

4.  FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga.

Authors:  Annemieke Aartsma-Rus; Arthur M Krieg
Journal:  Nucleic Acid Ther       Date:  2016-12-08       Impact factor: 5.486

5.  Biochemical characterization of patients with in-frame or out-of-frame DMD deletions pertinent to exon 44 or 45 skipping.

Authors:  Karen Anthony; Virginia Arechavala-Gomeza; Valeria Ricotti; Silvia Torelli; Lucy Feng; Narinder Janghra; Giorgio Tasca; Michela Guglieri; Rita Barresi; Annarita Armaroli; Alessandra Ferlini; Katherine Bushby; Volker Straub; Enzo Ricci; Caroline Sewry; Jennifer Morgan; Francesco Muntoni
Journal:  JAMA Neurol       Date:  2014-01       Impact factor: 18.302
  5 in total
  7 in total

Review 1.  Precision medicine for cardiovascular disease : Learning lessons from cardiomyopathies.

Authors:  F Sedaghat-Hamedani; H A Katus; B Meder
Journal:  Herz       Date:  2018-03       Impact factor: 1.443

Review 2.  Dilated cardiomyopathy as the initial presentation of Becker muscular dystrophy: a systematic review of published cases.

Authors:  Gaspar Del Rio-Pertuz; Cristina Morataya; Kanak Parmar; Sarah Dubay; Erwin Argueta-Sosa
Journal:  Orphanet J Rare Dis       Date:  2022-05-12       Impact factor: 4.303

3.  Efficient exon skipping of SGCG mutations mediated by phosphorodiamidate morpholino oligomers.

Authors:  Eugene J Wyatt; Alexis R Demonbreun; Ellis Y Kim; Megan J Puckelwartz; Andy H Vo; Lisa M Dellefave-Castillo; Quan Q Gao; Mariz Vainzof; Rita C M Pavanello; Mayana Zatz; Elizabeth M McNally
Journal:  JCI Insight       Date:  2018-05-03

Review 4.  Cardiac Pathophysiology and the Future of Cardiac Therapies in Duchenne Muscular Dystrophy.

Authors:  Tatyana A Meyers; DeWayne Townsend
Journal:  Int J Mol Sci       Date:  2019-08-22       Impact factor: 5.923

5.  Medical management of muscle weakness in Duchenne muscular dystrophy.

Authors:  Sarah R Rivera; Sumit K Jhamb; Hoda Z Abdel-Hamid; Gyula Acsadi; John Brandsema; Emma Ciafaloni; Basil T Darras; Susan T Iannaccone; Chamindra G Konersman; Nancy L Kuntz; Craig M McDonald; Julie A Parsons; Carolina Tesi Rocha; Craig M Zaidman; Russell J Butterfield; Anne M Connolly; Katherine D Mathews
Journal:  PLoS One       Date:  2020-10-19       Impact factor: 3.240

Review 6.  Principles and correction of 5'-splice site selection.

Authors:  Florian Malard; Cameron D Mackereth; Sébastien Campagne
Journal:  RNA Biol       Date:  2022-01       Impact factor: 4.766

7.  Full-Length Dystrophin Restoration via Targeted Exon Addition in DMD-Patient Specific iPSCs and Cardiomyocytes.

Authors:  Rou Xiao; Miaojin Zhou; Peiyun Wang; Baitao Zeng; Lingqian Wu; Zhiqing Hu; Desheng Liang
Journal:  Int J Mol Sci       Date:  2022-08-16       Impact factor: 6.208
  7 in total

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