TISSUE-ENGINEERED SKELETAL-MUSCLE ORGANOIDS FOR REVERSIBLE GENE-THERAPY

Genetically modified murine skeletal myoblasts were tissue engineered in vitro into organ-like structures (organoids) containing only postmi totic myofibers secreting pharmacological levels of recombinant human growth hormone (rhGH). Subcutaneous organoid implantation under tensio n led to the rapid and stable appearance of physiological sera levels of rhGH for up to 12 weeks, whereas surgical removal led to its rapid disappearance. Reversible delivery of bioactive compounds from postmit otic cells in tissue engineered organs has several advantages over oth er forms of muscle gene therapy.