Gene transfer to muscle tissue

Gene transfer to muscle tissue" describes the process of delivering exogenous genetic material (such as plasmid DNA or viral gene therapy vectors) into muscle cells with the goal of achieving stable and often long-lasting expression of therapeutic proteins. This technique is primarily investigated for treating muscle disorders (such as muscular dystrophies), programming muscle to secrete systemic therapeutic proteins (e.g., erythropoietin, clotting factors), or for vaccination purposes[1][2][3][4][5]. The methods used for gene transfer to muscle include direct injection of 'naked' plasmid DNA, electroporation (using electrical pulses), and genetically engineered viral vectors, often with tissue tropism modified for skeletal muscle[1][2][3][5]. While muscle cells can take up and stably express foreign genes, no individual protein, receptor, or enzyme is universally required for the process, though molecules such as Myomaker and Myomerger are essential for muscle cell fusion and can affect efficiency in certain gene therapy vector designs[2]. This entry should not be considered a molecular target but rather a therapeutic strategy or platform. For actual molecular targets relevant to gene transfer in muscle (such as surface receptors facilitating viral entry), further specificity and context would be required.

Other names

Gene delivery to muscle tissueMuscle-directed gene therapy

Gene delivery and transgene expression in muscle fibers