Development of trofinetide for the treatment of Rett syndrome: from bench to bedside

• Main Authors: Melissa Kennedy (Author), Larry Glass (Author), Daniel G. Glaze (Author), Steve Kaminsky (Author), Alan K. Percy (Author), Jeffrey L. Neul (Author), Nancy E. Jones (Author), Daniela Tropea (Author), Joseph P. Horrigan (Author), Paige Nues (Author), Kathie M. Bishop (Author), James M. Youakim (Author)
• Format: Book
• Published: Frontiers Media S.A., 2024-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Rett syndrome (RTT) is rare neurodevelopmental disorder caused by mutations in the MECP2 gene that encodes methyl-CpG-binding protein 2 (MeCP2), a DNA-binding protein with roles in epigenetic regulation of gene expression. Functional loss of MeCP2 results in abnormal neuronal maturation and plastici...