NIH Reporter #5R01NS134923-03

Uncovering mechanisms and developing novel therapeutic strategies for TBCD-related developmental and epileptic encephalopathy

NIH awarded a research grant to study TBCD-related developmental and epileptic encephalopathy, focusing on disease mechanisms and novel therapeutic strategies including gene therapy, using organoid and mouse models.

Description

This is a NIH research grant opportunity focused on molecular and cellular mechanisms of TBCD-related encephalopathy, including development of gene therapy approaches. The project involves in vitro organoid models and in vivo mouse models, with a multidisciplinary approach to uncover disease mechanisms and evaluate therapeutic strategies.

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