Drug intelligence / Profile preview

MYOD1 retroviral vector

Development stage
Preclinical
Lead developer
National Center of Neurology and Psychiatry
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Ex Vivo
01

Overview

MYOD1 retroviral vector is a gene therapy tool designed to deliver the *MYOD1* (Myogenic Differentiation 1) gene into target cells, typically non-muscle cells like fibroblasts or urine-derived cells (UDCs). MYOD1 is a master transcription factor and a member of the basic helix-loop-helix (bHLH) family that initiates the myogenic program. By overexpressing MYOD1 via a retroviral vector, researchers can induce transdifferentiation of these cells into myotubes that express muscle-specific proteins such as myosin heavy chain and dystrophin. This technology is primarily utilized to create patient-specific *in vitro* models for Duchenne muscular dystrophy (DMD), enabling the study of disease pathogenesis and the evaluation of therapeutic strategies like antisense oligonucleotide-mediated exon skipping. The vector allows for a stable and reproducible platform to assess the efficacy of genetic corrections in a myogenic context.

Other names
MyoD retroviral vectorMyogenic differentiation 1 retroviral vectorMYOD1-transducing retroviral vectorMYOD-1-transducing retroviral vectorMYOD 1-transducing retroviral vector
02

Targets

DNAMYOD1 (Myoblast determination protein 1)

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