Optimized Human Regulatory Sequences Achieve Targeted Expression in CNS and Decreased Liver Expression in Mice

Mitchell Lopez, Serena X. Liu, Steven A. Tan, Tulasi I. Solanki, Ben Zhao,Puja Dhanota,Andrew Kim, Simon Levine,James S. Griffin,John McLaughlin, Gregory Lucey, Kenneth McCullough, Saki Shimizu,Chi Zhang, Maria Candidia Vila, Kartik Ramamoorthi, Stephanie Tagliatela, Raghavendra Hosur,Annie Tanenhaus,Martin Moorhead

MOLECULAR THERAPY(2022)

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