Thomas E. Jensen
Using temperature-sensitive liposomes to localize drug-treatment of neuromuscular disorders
Thermosensitive liposomes (TSLs) are nanosized lipid spheres designed to release an encapsulated drug when heated to ~42°C. TSLs in combination with localized heating enable targeted drug release in specific bodily tissues. Neuromuscular junctions (NMJs) are synapses between motor neurons and skeletal muscle cells, and impaired NMJ function contributes to neuromuscular disease (NMD).
Several drug candidates have been shown to prevent or treat NMD in animals, but their widespread effects in tissues other than skeletal muscle preclude their use in humans. Theoretically, TSL technology could target the effects of broadly acting drugs onto skeletal muscles and their NMJs.
This project will investigate in mice whether TSLs combined with localized heating can be used to confine the effects of drugs to skeletal muscles. The perspective is to treat a wide range of conditions characterized by loss of muscle and NMJ function, ranging from physical inactivity during hospitalization, age-related sarcopenia, and cachexia to various genetic NMDs.
