SMA Type 1: How Gene Therapy Works
Автор: NationwideChildrens
Загружено: 2017-12-07
Просмотров: 192895
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A one-time intravenous infusion of the high dose of gene therapy extended the survival of patients with spinal muscular atrophy type 1 (SMA1) in a Phase 1 clinical trial, according to a study published Nov. 1, 2017 in the New England Journal of Medicine. This animation describes how the process works.
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