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Scientist whose mother and sisters died of ALS complications hopes experimental treatment will save his life - CBS News
Overview
Jeff Vierstra has received repeated spinal infusions designed to disable a mutated ALS gene as an experimental preventive therapy; his case highlights the urgent personal stakes for patients pursuing novel genetic treatments. The positive results from the AAV1-hOTOF trial for inherited deafness show that gene-based therapies can reach human trials with demonstrable safety and functional benefit, underscoring wider progress but also the continuing need to study long-term effects, age-related responses, and predictive biomarkers across different genetic neurological conditions.
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ORGCBS NewsPERSONJeff VierstraPERSONJeff Vierstra's mother
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