Clinical-grade intranasal NGF fuels neurological and metabolic functions of Mecp2-deficient mice.

ABSTRACT: MECP2 deficiency causes a broad spectrum of neuropsychiatric disorders that can affect both genders. Rett syndrome is the most common and is characterized by an apparently normal growth period followed by a regression phase in which patients lose most of their previously acquired skills. After this dramatic period, various symptoms progressively appear, including severe intellectual disability, epilepsy, apraxia, breathing abnormalities and motor deterioration. MECP2 encodes for an epigenetic transcription factor that is particularly abundant in the brain; consequently, several transcriptional defects characterize the Rett syndrome brain. The well-known deficiency of several neurotrophins and growth factors, together with the positive effects exerted by trofinetide, a synthetic analogue of

SUBMITTER: Pozzer D 

PROVIDER: S-EPMC11884770 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature