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.
Journal article
2023-04-01T00:00:00+00:00
4
220 - 222
2
Paris-Saclay University, UVSQ, INSERM-UMR1179, END-ICAP Laboratory, 78000 Versailles, France; SQY Therapeutics, 78180 Montigny-Le-Bretonneux, France.
Humans, Muscular Dystrophy, Duchenne, Dystrophin, Utrophin, Up-Regulation, Patient Acuity