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Updated: Feb 14, 2026

Biotin-based Pulldown Assay to Validate mRNA Targets of Cellular miRNAs
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Targeted mRNA Therapy for Ornithine Transcarbamylase Deficiency

Mary G Prieve1, Pierrot Harvie1, Sean D Monahan1

  • 1PhaseRx, Inc., 410 W. Harrison Street, Suite 300, Seattle, WA 98119, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|February 14, 2018
PubMed

Keywords:
LNPOTCDOtc(spf -ash) micehybrid mRNA technology delivery systemi-ERTinherited metabolic disorderintracellular enzyme replacement therapylipid nanoparticlemRNA deliveryornithine transcarbamylase deficiency

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