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Human Gene Therapy|January 11, 2018
AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease)Allison M Bradbury, Mohammed A Rafi, Jessica H Bagel, et al.The Journal of Clinical Investigation|August 11, 2020
Krabbe disease successfully treated via monotherapy of intrathecal gene therapyAllison M Bradbury, Jessica H Bagel, Duc Nguyen, et al.Pageof 8