New gene therapy targeting C9orf72-ALS begins Phase 1 clinical trial in the UK | Dr Keith Mayl & Dr Ahmad Al Khleifat | MND Research Blog

Researchers at King’s College Hospital, led by Professor Christopher Shaw, have embarked on the first gene therapy clinical trial for ALS patients with a C9orf72 gene mutation. Since the mutated gene produces toxic products, blocking the gene with gene therapy might be a useful approach to treatment.  Click here to read the full article

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