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Taghian, Toloo; Marosfoi, Miklos G.; Puri, Ajit S.; Cataltepe, Oguz.I.; King, Robert M.; Diffie, Elise B.; Maguire, Anne S.; Martin, Douglas R.; Fernau, Deborah; Batista, Ana Rita; Kuchel, Tim; Christou, Chris; Perumal, Raj; Chandra, Sundeep; Gamlin, Paul D.; Bertrand, Stephanie G.; Flotte, Terence R.; McKenna-Yasek, Diane; Tai, Phillip W.L.; Aronin, Neil; Gounis, Matthew J.; Sena-Esteves, Miguel; Gray-Edwards, Heather L.
Molecular therapy, 02/2020, Letnik: 28, Številka: 2Journal Article
Global gene delivery to the CNS has therapeutic importance for the treatment of neurological disorders that affect the entire CNS. Due to direct contact with the CNS, cerebrospinal fluid (CSF) is an attractive route for CNS gene delivery. A safe and effective route to achieve global gene distribution in the CNS is needed, and administration of genes through the cisterna magna (CM) via a suboccipital puncture 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 of an intravascular microcatheter, which can be safely navigated intrathecally under fluoroscopic guidance. We examined the safety, reproducibility, and distribution/transduction of this method in sheep using a self-complementary adeno-associated virus 9 (scAAV9)-GFP vector. This technique was used to treat two Tay-Sachs disease patients (30 months old and 7 months old) with AAV gene therapy. No adverse effects were observed during infusion or post-treatment. This delivery technique is a safe and minimally invasive alternative to direct infusion into the CM, achieving broad distribution of AAV gene transfer to the CNS. Cisterna magna injection of AAV gene therapy results in global distribution but is risky in humans. Taghian et al. describe a microcatheter-mediated technique for cisterna magna injection that results in widespread scAAV9-GFP distribution in sheep. This technique was then used to deliver AAV in two children with Tay-Sachs disease.
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