Intracisternal Delivery to the CNS of an AAV Gene Therapy Candidate for the Treatment of GM1 Gangliosidosis

Time: 8:40 am
day: Day One


  • Routes of delivery of AAV vectors to the CNS
  • Intracisternal (ICM) administration: bioavailability and safety considerations
  • Preclinical evaluation of ICM-administered AAVrh10 vector carrying human GLB1
  • Status of clinical trial in GM1 gangliosidosis patients