The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy

The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy

Abstract During the last decade, multiple clinical trials for Duchenne muscular dystrophy (DMD) have focused on the induction of dystrophin expression using different strategies. Many of these trials have reported a clear increase in dystrophin protein following treatment. However, the low levels of...

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Bibliographic Details
Main Authors: Dominic Scaglioni, Francesco Catapano, Matthew Ellis, Silvia Torelli, Darren Chambers, Lucy Feng, Matthew Beck, Caroline Sewry, Mauro Monforte, Shawn Harriman, Erica Koenig, Jyoti Malhotra, Linda Popplewell, Michela Guglieri, Volker Straub, Eugenio Mercuri, Laurent Servais, Rahul Phadke, Jennifer Morgan, Francesco Muntoni
Format: Article
Language:English
Published: BMC 2021-01-01
Series:Acta Neuropathologica Communications
Subjects:
Dystrophin
Muscular dystrophy
Immunofluorescence
Genetic therapies
Golodirsen
Clinical trial
Online Access:https://doi.org/10.1186/s40478-020-01106-1

Internet

https://doi.org/10.1186/s40478-020-01106-1

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