Utrophin correlates with disease severity in Duchenne muscular dystrophy.
Med
; 4(4): 220-222, 2023 04 14.
Article
in En
| MEDLINE
| ID: mdl-37060897
ABSTRACT
This month in Med, the description of an unusually severely affected DMD patient suffering from a large deletion in the dystrophin gene confirms that absence of utrophin worsens the dystrophy and supports the concept that utrophin upregulation ameliorates the pathology. This study may guide the development of dystrophin-based gene therapies.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Muscular Dystrophy, Duchenne
Type of study:
Diagnostic_studies
Aspects:
Patient_preference
Limits:
Humans
Language:
En
Journal:
Med
Year:
2023
Document type:
Article
Affiliation country: