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

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 the indu...

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Vydáno v:Acta Neuropathol Commun
Hlavní autoři: Scaglioni, Dominic, Catapano, Francesco, Ellis, Matthew, Torelli, Silvia, Chambers, Darren, Feng, Lucy, Beck, Matthew, Sewry, Caroline, Monforte, Mauro, Harriman, Shawn, Koenig, Erica, Malhotra, Jyoti, Popplewell, Linda, Guglieri, Michela, Straub, Volker, Mercuri, Eugenio, Servais, Laurent, Phadke, Rahul, Morgan, Jennifer, Muntoni, Francesco
Médium: Artigo
Jazyk:Inglês
Vydáno: BioMed Central 2021
Témata:
Research
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7789286/
https://ncbi.nlm.nih.gov/pubmed/33407808
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s40478-020-01106-1
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