Molecular Therapy

Medical research

Gene therapy generates new neurons to treat Huntington's disease

Huntington's disease (HD) is a rare disease characterized by abnormal chorea movement and caused by the Huntingtin (Htt) gene mutation and neurodegeneration in a brain area called the striatum. A research group led by Dr. ...

Neuroscience

Gene therapy helps functional recovery after stroke

A new gene therapy turns glial cells—abundant support cells in the brain—into neurons, repairing damage that results from stroke and significantly improving motor function in mice. A paper describing the new therapy, ...

Medical research

First in vivo proof-of-concept in Steinert's myotonic dystrophy

Ana Buj Bello's team, a researcher in an Inserm unit at Genethon, the AFM-Telethon laboratory, has made the proof-of-concept of a CRISPR-Cas9 approach in a mouse model of Steinert's myotonic dystrophy, the most common neuromuscular ...

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