An Engineered Galactosylceramidase Construct Improves AAV Gene Therapy for Krabbe Disease in Twitcher Mice
Human Gene Therapy（2019）
关键词
adeno-associated virus,AAV9,Krabbe disease,twitcher mice,systemic delivery,galactosylceramidase,globoid cell leukodystrophy,lysosomal storage disease,gene therapy,demyelination,life span
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