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Stem cell-gene therapy shows promise in ALS safety trial

September 5, 2022

Cedars-Sinai investigators have developed an investigational therapy using support cells and a protective protein that can be delivered past the blood-brain barrier. This combined stem cell and gene therapy can potentially protect diseased motor neurons in the spinal cord of patients with amyotrophic lateral sclerosis, a fatal neurological disorder known as ALS or Lou Gehrig’s disease.

In the first trial of its kind, the Cedars-Sinai team showed that delivery of this combined treatment is safe in humans.

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