ArticleFrontiers in medicine2026
rAAV9 vector biodistribution in nonhuman primate brain and spinal cord following lumbar intrathecal infusion.
Article in Frontiers in medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Introduction: Delivery of recombinant adeno-associated virus serotype 9 (rAAV9) into cerebrospinal fluid bypasses the blood-brain barrier and can permit efficient brain tissue transduction. Whereas intracisterna magna injection (ICM) and other routes have been considered, lumbar intrathecal (IT) delivery offers a minimally invasive approach for treating genetic disorders of the central nervous system. To evaluate central nervous system biodistribution, we quantified vector genome (vg) copies of five rAAV9 vectors in cynomolgus macaques following ICM or lumbar IT delivery. Methods: A total of 48 animals were followed across five studies: four lumbar IT infusion studies with four recombinant adeno-associated virus serotype 9 vectors (TSHA-101, -102, -105, and -120); and one ICM infusion study (TSHA-102 only). Stock vectors were diluted to dosing concentrations and 2.5 mL were administered to each animal. After animals were sacrificed, tissue samples were harvested and biodistribution was assessed via qPCR. Results: Despite protocol differences specifying different doses (human effective dose range, 2.6 × 10 Conclusion: These findings support IT administration as an effective, minimally invasive approach to central nervous system-directed gene therapy.
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