Next-generation sequencing provides novel insights into the mechanisms underlying autism and myotonic muscular dystrophy
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 Nationwide Children's Hospital pioneers new gene therapy for Duchenne muscular dystrophyNationwide Children's Hospital pioneers new gene therapy for Duchenne muscular dystrophy
 
In a landmark moment for the Abigail Wexner Research Institute at Nationwide Children's, a 5-year-old from Bellefontaine, Ohio, received the first dose of a recently approved gene therapy for Duchenne muscular dystrophy at Nationwide Children's Hospital, where the therapy was invented and initially tested.
 
 
 Next-generation sequencing provides novel insights into the mechanisms underlying autism and myotonic muscular dystrophyNext-generation sequencing provides novel insights into the mechanisms underlying autism and myotonic muscular dystrophy
 
The pathomechanistic connection between autism and myotonic muscular dystrophy type 1.
 
   Dual CRISPR-Cas3 is a promising tool to induce a gigantic genomic deletion and restore dystrophin proteinDual CRISPR-Cas3 is a promising tool to induce a gigantic genomic deletion and restore dystrophin protein
 
Researchers evaluated the use of a dual clustered regularly interspaced short palindromic repeats (CRISPR)-Cas3 system.
 
 Duchenne and other dystrophinopathies research receives major boost from new collaboration
 
Duchenne and other dystrophinopathies research receives major boost from new collaborationCritical Path Institute's (C-Path) Duchenne Regulatory Science Consortium (D-RSC) and the Duchenne Data Foundation (DDF) are excited to announce a joint collaboration aimed at advancing research and improving healthcare for individuals with Duchenne muscular dystrophy (DMD) and other dystrophinopathies (conditions linked to mutations in the DMD gene).
 
 
 Gene therapy shows promise for muscular dystrophy patients
 
Gene therapy shows promise for muscular dystrophy patientsResearchers at Cincinnati Children’s Hospital reveal how damage to the cell’s energy factory causes muscle wasting. Closing a pore in the mitochondrial membrane in gene-edited mice prevents disease progression.