Genetically corrected human stem cells help mice with a disease resembling limb-girdle MD; cell membrane sealants are being studied in Duchenne and other forms of muscular dystrophy
LGMD2D mice benefit from corrected human stem cells
A multinational team of scientists successfully transplanted genetically corrected muscle stem cells derived from people with type 2D limb-girdle muscular dystrophy (LGMD2D) into LGMD2D research mice and saw better muscle function in these mice than in similar mice that didn't receive the cells.
Longtime MDA research grantee honored for work in LGMD gene therapy
A team headed by neurologist Jerry Mendell, a longtime MDA research grantee and director of the MDA Clinic at Nationwide Children's Hospital in Columbus, Ohio, has received the prestigious Annals of Neurology prize for an outstanding contribution to clinical neuroscience.
Five of six people with limb-girdle muscular dystrophy type 2D produced protein from the new genes
Results from an MDA-supported, phase 1 study of gene therapy for the type 2D form of limb-girdle muscular dystrophy (LGMD2D) show sustained protein production from the transferred genes in two out of three trial participants six months after injection of the genes into a foot muscle.
The new results follow an announcement last year showing protein production from transferred genes in the first...
Featured in this issue: Duchenne muscular dystrophy exon skipping trial ** Idebenone in DMD ** Gene injections for type 2D limb girdle muscular dystrophy ** Patient registry for LGMD2B and Miyoshi myopathy
The following article contains items about: Duchenne and limb-girdle muscular dystrophies