Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues-Reference-Cited by-同舟云学术

Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues

Published:2017-10 Issue:5 Volume:27 Page:251-259
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

Aartsma-Rus Annemieke¹²,Straub Volker²,Hemmings Robert³,Haas Manuel⁴,Schlosser-Weber Gabriele⁵,Stoyanova-Beninska Violeta⁶,Mercuri Eugenio⁷⁸,Muntoni Francesco⁹,Sepodes Bruno¹⁰,Vroom Elizabeth¹¹,Balabanov Pavel⁴

Affiliation:

1. Department of Human Genetics, Leiden University Medical Center, Leiden, the Netherlands.

2. John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.

3. Medicines and Healthcare Product Regulatory Agency, London, United Kingdom.

4. Central Nervous System and Ophthalmology, Scientific and Regulatory Management Department, Human Medicines Evaluation Division, European Medicines Agency, London, United Kingdom.

5. Bundesinstitut für Arzneimittel und Medizinprodukte, Bonn, Germany.

6. Medicines Evaluation Board, Utrecht, the Netherlands.

7. Department of Pediatric Neurology, Catholic University, Rome, Italy.

8. Centro Clinico Nemo, Policlinico Gemelli, Rome, Italy.

9. Dubowitz Neuromuscular Center, UCL Great Ormond Street Institute of Child Health, London, United Kingdom.

10. Faculdade de Farmácia, Universidade de Lisboa, Lisboa, Portugal.

11. United Parent Project Muscular Dystrophy, Amsterdam, the Netherlands.

Publisher

Mary Ann Liebert Inc

Subject

Drug Discovery,Genetics,Molecular Biology,Molecular Medicine,Biochemistry

Link