Reversibility and therapeutic feasibility of DNM1L-associated neurodevelopmental disorders.

Abstract excerpt

DNM1L-associated encephalopathy is a neurological disorder with a broad spectrum of symptoms associated with mutations in the DNM1L gene. Treatment primarily aims to alleviate symptoms, which is mostly ineffective as the underlying neuropathology is not well understood. Moreover, the progression and reversibility of the molecular pathology across the key developmental and postnatal stages have not been well...

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