ERT Degrades Gene Therapy for Storage Disorder.
作者信息
Markusic David Michael
机构信息
Assistant Research Professor of Pediatrics, Herman B. Wells Center for Pediatric Research, 1044 W. Walnut Street, R4-171, Indianapolis, IN 46202, USA.
出版信息
Mol Ther. 2019 Jul 3;27(7):1207-1208. doi: 10.1016/j.ymthe.2019.06.002. Epub 2019 Jun 13.
Abstract
摘要
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3
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