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Research Interests

Papers共 77 篇Author StatisticsCo-AuthorSimilar Experts

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Community consensus for Heparan sulfate as a biomarker to support accelerated approval in Neuronopathic Mucopolysaccharidoses
Joseph Muenzer,Carole Ho,Heather Lau, Mark Dant,Maria Fuller, Nidal Boulos,Patricia Dickson, N. Matthew Ellinwood,Simon A. Jones, Eric Zanelli, Cara O'Neill
Molecular Genetics and Metabolismno. 4 (2024): 108535-108535
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First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B
Paul Harmatz, Carlos E. Prada,Barbara K. Burton,Heather Lau,Craig M. Kessler,Liching Cao,Marina Falaleeva, Andres G. Villegas, Jennifer Zeitler, Kathleen Meyer,Weston Miller, Cheryl Wong Po Foo,
Molecular Therapyno. 12 (2022): 3587-3600
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