Researchers combine gene therapy with optogenetics to curb abnormal neural activity and prevent seizure-like activity in neurons.

The U.S. Food and Drug Administration approved PTC Therapeutics, Inc.'s Kebilidi (eladocagene exuparvovec-tneq) gene therapy for the ...

The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus ...

This study presents a valuable finding on the molecular mechanisms that govern GABAergic inhibitory synapse function. The authors propose that Endophilin A1 serves as a novel regulator of GABAergic ...

KEBILIDI is an adeno-associated virus vector-based gene therapy indicated for the treatment ... KEBILIDI is contraindicated in patients who have not achieved skull maturity assessed by neuroimaging.

First-ever FDA approval for gene therapy directly administered to the brain - - Priority review voucher granted - - Broad label including children and adults - - ...

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This is the first gene therapy for AADC deficiency to receive US regulatory approval and the first that can be administered directly to the brain.

This valuable study investigates how the neural representation of individual finger movements changes during the early period of sequence learning. By combining a new method for extracting features ...

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