Development of NanOVEctors for Lysosomal-based therapeutic strategies to combat lysosomal-related neurodegenerative diseases – NOVEL

Neurodegenerative diseases represent some of the greatest challenges for basic science and clinical medicine because of their prevalence, cost, complex biochemistry and pathology, and lack of mechanism-based treatments. Genetic and neuropathological evidence indicates that a likely mechanism linking these diseases relates to a lysosomal impairment, making it a point of particular vulnerability. Additionally, a major challenge in therapeutic drug delivery so far lies in reaching the target neurons and impaired intracellular organelles. The development of nanotechnology-based drug delivery systems for drugs intended to treat brain diseases is a promising avenue because nanovectors offer the ability to carry drugs to the sick cells, and the prolonged drug release profile possible with these systems permits longer action. The NOVEL aim is to develop dual-targeting nanosystem-based therapies for restoring lysosomal function and activity.