Translational Gene Therapy Approaches to Treat Mucopolysaccharidosis

Time: 11:15 am
day: Day One


  • Discussing the potentiality of intracerebrospinal fluid adenoassociated viral (AAV) vector-mediated gene therapy to counteract neurologic and somatic MPS
  • Using this approach to treat for MPSII and MPSIII, expression of the different therapeutic genes was detected in widespread brain regions and in the liver, leading to increased enzyme activity in CNS and serum and simultaneous correction of both central and somatic disease
  • The results of these studies provide strong evidence supporting the clinical translation of the approach not only for MPS but also for other genetic diseases that course with neurodegeneration