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…

Potential Gene Therapy for Rett Syndrome in the Works

A gene therapy aimed at slowing disease progression and improving movement, coordination and communication in children with Rett syndrome, is being developed. Mostly seen in girls, Rett affects brain development, causing impairments such as progressive loss of motor skills and speech. “It’s often a full-time job as a parent,…

Study Identifies Potential Blood-based Biomarkers, Paths for Treatment

Researchers have identified a set of RNA molecules that are dysregulated in Rett syndrome and may be relevant biomarkers for the disorder. The study, “Unraveling Molecular Pathways Altered in MeCP2-Related Syndromes, in the Search for New Potential Avenues for Therapy,” was published in the journal…