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Molecular Therapy : the Journal of the American Society of Gene Therapy|February 15, 2026
Focused ultrasound expands intra-CSF AAV delivery to deep brain regions in rats and non-human primatesRikke Hahn Kofoed, Bradford Elmer, Kate Noseworthy, et al.Science (New York, N.Y.)|November 3, 2018
Poly(ADP-ribose) drives pathologic α-synuclein neurodegeneration in Parkinson's diseaseTae-In Kam, Xiaobo Mao, Hyejin Park, et al.The Journal of Clinical Investigation|June 19, 2025
Cross-species efficacy of AAV-mediated ARSA replacement for metachromatic leukodystrophyShyam Ramachandran, Jeffery Ardinger, Jie Bu, et al.Science Translational Medicine|July 29, 2021
PARIS farnesylation prevents neurodegeneration in models of Parkinson's diseaseAreum Jo, Yunjong Lee, Tae-In Kam, et al.Pageof 2