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Yale Study Reveals Key Gene in Lissencephaly Treatment

1/5/2025

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Summary

Story summary

A groundbreaking Yale study has unveiled a molecular mechanism behind lissencephaly, a rare brain disorder that triggers seizures and intellectual disabilities. Researchers pinpointed a gene associated with the condition and discovered that a drug activating the mTOR pathway could prevent and even reverse brain malformations in organoids, paving the way for promising treatments for different lissencephaly types.

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Story summary

A groundbreaking Yale study has unveiled a molecular mechanism behind lissencephaly, a rare brain disorder that triggers seizures and intellectual disabilities. Researchers pinpointed a gene associated with the condition and discovered that a drug activating the mTOR pathway could prevent and even reverse brain malformations in organoids, paving the way for promising treatments for different lissencephaly types.