genetically corrected autologous mesoangioblasts

Genetically corrected autologous mesoangioblasts are an experimental cell-based gene therapy for Duchenne muscular dystrophy (DMD). Mesoangioblasts are vessel-associated progenitor cells capable of differentiating into skeletal muscle. In this autologous approach, mesoangioblasts are isolated from the patient's own muscle biopsy, expanded in vitro, and genetically modified—typically using a lentiviral vector—to express a functional version of the dystrophin protein (such as micro-dystrophin). The corrected cells are then delivered systemically via intra-arterial injection. A key advantage of mesoangioblasts over other muscle progenitors (like satellite cells) is their ability to cross the blood-vessel wall and migrate into the surrounding muscle tissue, potentially allowing for more widespread distribution. The therapy aims to restore dystrophin expression, stabilize muscle fibers, and improve motor function in DMD patients.

Other names

autologous mesoangioblasts expressing dystrophingenetically modified autologous mesoangioblastsLenti-MABs