Journal
HUMAN GENE THERAPY METHODS
Volume 29, Issue 5, Pages 212-219Publisher
MARY ANN LIEBERT, INC
DOI: 10.1089/hgtb.2018.041
Keywords
intrathecal; central nervous system; AAV; gene therapy
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Intrathecal delivery of adeno-associated virus vectors and other therapeutics are currently being evaluated for the treatment of central nervous system sequelae of lysosomal storage diseases, motor neuron diseases, and neurodegenerative diseases. As products transition from preclinical to clinical studies, a standardized and clinically relevant method of intrathecal delivery is increasingly germane. Here, we describe a method of intrathecal delivery via suboccipital puncture into the cisterna magna under fluoroscopic guidance in nonhuman primates. This procedure is suitable for use in good laboratory practice compliant studies, has an excellent safety profile, and is highly similar to the procedure currently being explored for use in humans.
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