hSynC-Dp427m

hSynC-Dp427m is an experimental ex vivo gene therapy candidate designed for the treatment of Duchenne Muscular Dystrophy (DMD). It utilizes the human synthetic chromosome (hSynC) platform, a non-integrating vector system capable of carrying large genetic payloads that exceed the capacity of traditional viral vectors like adeno-associated virus (AAV). The construct delivers the full-length human dystrophin gene (DMD) cDNA, specifically the Dp427m isoform, which is approximately 14 kb in size. By providing the complete coding sequence, hSynC-Dp427m aims to restore the expression of functional, full-length dystrophin protein rather than the truncated micro-dystrophin versions used in AAV-based therapies. The platform is engineered with a genetic safety switch to mitigate potential adverse effects and is intended for use with cell-based delivery methods, such as induced pluripotent stem cells (iPSCs). Preclinical studies involving xenotransplantation of human cells containing the hSynC-Dp427m construct into mice have confirmed successful expression of human dystrophin.

Other names

human synthetic chromosome-Dp427m