Translational and Regulatory Challenges for Exon Skipping Therapies-Reference-Cited by-同舟云学术

Translational and Regulatory Challenges for Exon Skipping Therapies

Published:2014-10 Issue:10 Volume:25 Page:885-892
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Aartsma-Rus Annemieke¹²,Ferlini Alessandra³,Goemans Nathalie⁴,Pasmooij Anna M.G.⁵⁶,Wells Dominic J.⁷,Bushby Katerine²,Vroom Elizabeth⁸,Balabanov Pavel⁹

Affiliation:

1. Department of Human Genetics, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.

2. Institute of Genetic Medicine, Newcastle University, NE1 3BZ Newcastle, United Kingdom.

3. Department of Medical Sciences, University of Ferrara, 44121 Ferrara, Italy.

4. Child Neurology, University Hospitals Leuven, B-3000 Leuven, Belgium.

5. Dutch Medicines Evaluation Board, 3531 AR Utrecht, The Netherlands.

6. Department of Dermatology, University Medical Center Groningen, University of Groningen, 9700 RB Groningen, The Netherlands.

7. Department of Comparative Biomedical Sciences, Royal Veterinary College, London NW1 0TU, United Kingdom.

8. Duchenne Parent Project, 3905 PG Veenendaal, The Netherlands.

9. Scientific and Regulatory Management Department, European Medicines Agency, Central Nervous System and Ophthalmology Office, London E14 5EU, United Kingdom.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2014.086

Reference36 articles.

1. Antisense-mediated modulation of splicing: Therapeutic implications for Duchenne muscular dystrophy

2. Antisense-Mediated Exon Skipping: Networking to Meet Opportunities and to Overcome Challenges

3. Dystrophin Analysis in Clinical Trials

4. Antisense-Induced Multiexon Skipping for Duchenne Muscular Dystrophy Makes More Sense

Cited by 39 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Duchenne muscular dystrophy: disease mechanism and therapeutic strategies;Frontiers in Physiology;2023-06-26

2. Treatment and Management of Muscular Dystrophies;Neuromuscular Disorders;2022

3. Restoring Protein Expression in Neuromuscular Conditions: A Review Assessing the Current State of Exon Skipping/Inclusion and Gene Therapies for Duchenne Muscular Dystrophy and Spinal Muscular Atrophy;BioDrugs;2021-06-07

4. A Multifunctional LNA Oligonucleotide-Based Strategy Blocks AR Expression and Transactivation Activity in PCa Cells;Molecular Therapy - Nucleic Acids;2021-03

5. Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies;Expert Opinion on Investigational Drugs;2021-01-06