Standardized Method for Intra-Cisterna Magna Delivery Under Fluoroscopic Guidance in Nonhuman Primates

Human Gene Therapy Methods
Nathan KatzJames M Wilson

Abstract

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.

References

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Citations

Mar 1, 2019·British Journal of Pharmacology·Lars M IttnerYazi D Ke
Sep 17, 2020·Annals of Clinical and Translational Neurology·Christian HindererJames M Wilson
Aug 28, 2020·Human Gene Therapy·Juliette HordeauxJames M Wilson
Dec 10, 2019·Molecular Therapy : the Journal of the American Society of Gene Therapy·Toloo TaghianHeather L Gray-Edwards
May 19, 2020·Molecular Therapy. Methods & Clinical Development·Christian HindererJames M Wilson

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Methods Mentioned

BETA
X-ray
sedation

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