Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by dystrophin
deficiency. Dystrophin consists of the amino terminus, central rod domain with 24 spectrin …

CRISPR editing of muscle stem cells (MuSCs) with adeno-associated virus serotype-9
(AAV9) holds promise for sustained gene repair therapy for muscular dystrophies. However …

Duchenne muscular dystrophy (DMD) is a devastating and debilitating muscle degenerative
disease affecting 1 in every 3,500 male births worldwide. DMD is progressive and fatal; …

Sequencing of the human genome and numerous advances in molecular techniques have
launched the era of genetic medicine. Increasingly precise technologies for genetic …

Genome editing technology is a technique for targeted genetic modifications, enabling the
knockout and addition of specific DNA fragments. This technology has been widely used in …

Representatives of academia, patient organisations, industry and the United States Food
and Drug Administration attended a workshop on dystrophin quantification methodology …

Introduction Exon skipping compounds restoring the dystrophin transcript reading frame
have received regulatory approval for Duchenne muscular dystrophy (DMD). Recently, focus …

Duchenne muscular dystrophy (DMD) is an intractable genetic muscular disorder
characterized by the loss of DYSTROPHIN. The restoration of DYSTROPHIN is expected to …

Background This exploratory case-control study is to evaluate the effects of supplementation
of Aureobasidium pullulans-N-163 strain produced 1, 3–1,− 6 beta glucan in young patients …

In a phase 1/2, open-label dose escalation trial, we delivered rAAVrh74. MCK. GALGT2
(also B4GALNT2) bilaterally to the legs of two boys with Duchenne muscular dystrophy …