μDysCT194

μDysCT194 is a novel, optimized human microdystrophin transgene variant featuring an extended C-terminal (CT) domain (specifically containing 194 amino acids of the CT domain, including α- and β-syntrophin binding sites and Helix 1 of the coiled-coil domain). It is the active transgene component of pivodisgene parvec (RGX-202), an investigational adeno-associated virus serotype 8 (AAV8) vector-based gene therapy developed by REGENXBIO for the treatment of Duchenne muscular dystrophy (DMD). Unlike other clinical-stage microdystrophins that lack the C-terminal domain, μDysCT194 is designed to recruit key signaling proteins of the dystrophin-associated protein complex (DAPC)—such as α-syntrophin, α-dystrobrevin, and neuronal nitric oxide synthase (nNOS)—to the sarcolemma. This recruitment enhances sarcolemmal integrity, improves muscle resistance to contraction-induced damage, and supports muscle repair.

Other names

ABD1-H1-R1-R2-R3-H3-R24-H4-CR-CT194ABD-1-H1-R1-R2-R3-H3-R24-H4-CR-CT194ABD 1-H1-R1-R2-R3-H3-R24-H4-CR-CT194muDysCT194muDysCT-194muDysCT 194pivodisgene parvecuDysCT194uDysCT-194uDysCT 194