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A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates

Dan Wang, Shaoyong Li, Dominic J. Gessler and 12 more

Molecular Therapy: Methods & Clinical Development · 2018

doi:10.1016/j.omtm.2018.03.004

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