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A New Therapeutic Window for Patients with Genetic Neuropathy Disorders
Автор: Gladstone Institutes
Загружено: 2023-09-12
Просмотров: 4165
Описание:
Saisha is a charismatic 8-year-old girl with Charcot-Marie-Tooth disease (CMT), a progressive disorder that causes damage to nerves in the arms and legs that control movement, slowly reducing her ability to walk and use her hands.
She and her family visited Gladstone Institutes and the lab of Bruce Conklin, whose team is working on a gene editing therapy that could help her. They share their story and hopes for the future.
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