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MyoD lentiviral vector is a gene therapy research tool designed to deliver the MYOD1 (Myoblast Determination Protein 1) gene into non-muscle cells, such as fibroblasts and urinary stem cells (USCs). MYOD1 is a master transcriptional regulator that initiates the skeletal muscle differentiation program. By overexpressing this factor, the vector induces myogenic reprogramming, converting target cells into myoblasts and multinucleated myotubes in vitro. This technology is primarily utilized to create patient-specific cellular models for neuromuscular disorders, most notably Duchenne muscular dystrophy (DMD). These models allow researchers to investigate disease mechanisms and evaluate the efficacy of therapeutic candidates, such as antisense oligonucleotides, without the need for invasive muscle biopsies.
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