Potential New Therapeutic Targets Identified in Preclinical Study

Potential New Therapeutic Targets Identified in Preclinical Study

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Potential New Therapeutic Targets Identified in Preclinical Study

Targeting a biological pathway regulated by MECP2 — the gene mutated in most cases of Rett syndrome — normalized neural development in a study using mice, patient-derived cells, and a 3D model of the brain. The finding suggests that this pathway could become a therapeutic target in Rett syndrome. The study, “MeCP2 controls neural stem cell fate specification through miR-199a-mediated inhibition of BMP-Smad signaling,” was published in Cell Reports. Among its many roles, MeCP2, the protein made…

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