Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing

Title
Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing
Authors
Keywords
-
Journal
MOLECULAR THERAPY
Volume 26, Issue 4, Pages 1127-1136
Publisher
Elsevier BV
Online
2018-03-11
DOI
10.1016/j.ymthe.2018.03.002

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