Dystrophin mRNA Processing in the Canine Homologue of Duchenne Muscular Dystrophy: An Authentic Model for Gene Therapy

1992 ◽  
pp. 146-157
Author(s):  
N. J. H. Sharp ◽  
J. N. Kornegay ◽  
S. D. van Camp ◽  
M. H. Herbstreith ◽  
S. L. Secore ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Mrna Processing

scholarly journals Advances in gene therapy for muscular dystrophies

F1000Research ◽  
2016 ◽  
Vol 5 ◽  
pp. 2030 ◽  
Author(s):  
Hayder Abdul-Razak ◽  
Alberto Malerba ◽  
George Dickson
Keyword(s):  
Gene Therapy ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Gene Transcription ◽  
Therapeutic Strategies ◽  
Mrna Processing ◽  
Muscular Dystrophies ◽  
Gene Encoding ◽  
Recessive Lethal ◽  
Cellular Machinery

Duchenne muscular dystrophy (DMD) is a recessive lethal inherited muscular dystrophy caused by mutations in the gene encoding dystrophin, a protein required for muscle fibre integrity. So far, many approaches have been tested from the traditional gene addition to newer advanced approaches based on manipulation of the cellular machinery either at the gene transcription, mRNA processing or translation levels. Unfortunately, despite all these efforts, no efficient treatments for DMD are currently available. In this review, we highlight the most advanced therapeutic strategies under investigation as potential DMD treatments.

scholarly journals Myostatin Is a Quantifiable Biomarker for Monitoring Pharmaco-gene Therapy in Duchenne Muscular Dystrophy

2020 ◽  
Vol 18 ◽  
pp. 415-421 ◽  
Author(s):  
Virginie Mariot ◽  
Caroline Le Guiner ◽  
Inès Barthélémy ◽  
Marie Montus ◽  
Stéphane Blot ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy
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