The use of a shortened MeCP2 protein construct in Rett syndrome protein replacement therapy
Alexander Beribisky, Hannes Steinkellner, Claudia Sulek, Anna Huber, Victoria Sarne, Franco Laccone · FENS Forum 2024
Rett syndrome (RTT) is a severe neurodevelopmental disorder linked to abnormal levels of methyl CpG binding protein 2 (MeCP2), a global transcriptional regulator, known to both enhance and silence gene expression. It has been demonstrated on numerous occasions, however, that replenishing functional MeCP2 levels in the central nervous system, can ameliorate and even reverse the RTT phenotype. One such RTT treatment approach involves administering recombinantly purified MeCP2 while tethered to the cell-penetrating peptide TAT which is known to transduce fused protein cargo across cell membrane a
ePoster · Neuroscience