is needed, and administration of genes through the cisterna magna (CM) via a suboccipitalpuncture results in broad distribution in the brain and spinal cord. However, translation of this technique to clinical practice is challenging due to the risk of serious and potentially fatal complications in patients. Herein, we report development of a gene therapy delivery method to the CM through adaptation
are evaluating adeno-associated virus 9 for the treatment of neurodegenerative diseases using systemic or intrathecal lumbar administration. In large animals, administration via suboccipitalpuncture gives better brain transduction than lumbar administration. Here, we conducted a good laboratory practice-compliant investigational new drug-enabling study to determine the safety of suboccipital adeno-associated
-associated virus serotype 9 (AAV9) for the treatment of neurodegenerative diseases. Although these trials focus on systemic or lumbar administration, intrathecal administration via suboccipitalpuncture into the cisterna magna has demonstrated remarkable efficacy in large animals. We, therefore, conducted a good laboratory practice-compliant non-clinical study to investigate the safety of suboccipital AAV9
genetic and acquired diseases affecting the entire CNS. We characterized the transduction efficiency of two routes of vector administration into the CSF of cynomolgus macaques-lumbar puncture, which is typically used in clinical practice, and suboccipitalpuncture, which is more commonly used in veterinary medicine. We found that delivery of vector into the cisterna magna via suboccipitalpuncture is up