Medical research

Research toward healing the hearts of children with Duchenne

After a progressive weakening of the muscles takes away their motor skills, and then their abilities to stand and walk, most males with Duchenne muscular dystrophy die of heart and respiratory failure in their 20s.

Neuroscience

Scientists move closer to treatment for Huntington's disease

A new variant of the gene-editing CRISPR/Cas9 system is safer and more specific than versions previously used in early research towards a treatment for Huntington's disease, shows research published today in Frontiers in ...

Genetics

Gene therapy for rare form of blindness wins US approval

U.S. health officials on Tuesday approved the nation's first gene therapy for an inherited disease, a treatment that improves the sight of patients with a rare form of blindness. It marks another major advance for the emerging ...

Ophthalmology

Genetic treatment for blindness may soon be reality

Patients who had lost their sight to an inherited retinal disease could see well enough to navigate a maze after being treated with a new gene therapy, according to research presented today at AAO 2017, the 121st Annual Meeting ...

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