Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I

Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I

Mucopolysaccharidosis kind I (MPS I) is an inherited metabolic dysfunction brought on by deficiency of the lysosomal enzyme alpha-L-iduronidase (IDUA). The 2 present therapies [hematopoietic stem cell transplantation (HSCT) and enzyme replacement therapy (ERT)], are insufficiently efficient in addressing neurologic illness, partially as a result of lack of ability of lysosomal enzyme to cross the […]