Translational and Regulatory Challenges for Exon Skipping Therapies

Aartsma-Rus, A; Ferlini, A; Goemans, N; Pasmooij, A M G; Wells, D J; Bushby, K; Vroom, E; Balabanov, P

doi:10.1089/hum.2014.086

Translational and Regulatory Challenges for Exon Skipping Therapies

Aartsma-Rus, A; Ferlini, A; Goemans, N; Pasmooij, A M G; Wells, D J; Bushby, K; Vroom, E; Balabanov, P

Authors

A Aartsma-Rus

A Ferlini

N Goemans

A M G Pasmooij

D J Wells

K Bushby

E Vroom

P Balabanov

Citation

Aartsma-Rus, A., Ferlini, A., Goemans, N., Pasmooij, A. M. G., Wells, D. J., Bushby, K., …Balabanov, P. Translational and Regulatory Challenges for Exon Skipping Therapies. Human Gene Therapy, 25(10), 885-892. https://doi.org/10.1089/hum.2014.086

Journal Article Type	Article
Deposit Date	Nov 11, 2014
Publicly Available Date	Nov 21, 2020
Journal	HUMAN GENE THERAPY
Print ISSN	1043-0342
Publisher	Mary Ann Liebert
Volume	25
Issue	10
Pages	885-892
DOI	https://doi.org/10.1089/hum.2014.086
Public URL	https://rvc-repository.worktribe.com/output/1406260
Additional Information	Corporate Creators : Duchenne Parent Project, European Med Agency, Ferrara, Italy, Groningen, Katholieke Univ Leuven, Leiden Uni Medical Center, Newcastle