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Genetic therapies in store for neurodevelopmental disorders

EPISODE · Jun 16, 2025 · 44 MIN

Genetic therapies in store for neurodevelopmental disorders

from rare genetic disorder – ASF Weekly Science Podcasts

Gene therapies have been in the news lately. They are being used to help individuals who have a genetic variant linked to a disorder or disease, including but not limited to: spinal muscular atrophy, carbamoyl phosphate synthetase 1 (CPS1) deficiency, diabetes and some types of cancers. What is the promise in rare genetic forms of … Continue reading "Genetic therapies in store for neurodevelopmental disorders"

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Genetic therapies in store for neurodevelopmental disorders

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