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ExperimentPcsk9 adenine base editor efficiency in liver and nonliver tissue
Experiment ConditionBE-eVLP-Pcsk9
Assay Description: adenine base editing at target Pcsk9 site in liver vs. other mouse organs (heart, kidney, lung, muscle, spleen)
Tissue Measured:heart
Measurement Type:Editing Efficiency
Record ID15000001520

EditorTadA-8e V106W
Delivery SystemeVLP
Delivery System SubtypeVirus Like Particle
GuidePcsk9 Exon 1 splice domain
Vector

Model SpeciesMouse
Model NameC57BL/6J

Application Methodintravenous, retro-orbital
Application Siteliver
Dosage
Injection Frequencyonce
Injection Rate
Injection Volume120 microliters
Days post injection1 week
Editor Formatribonucleoprotein (RNP)
Antidote Id
Antidote Description

Measured Values
Samples Size: 4
 
Replicate Result (Editing Efficiency)
Measurement Units: % of total sequencing reads with AT converted to GC
Mean 0.39 
1 0.07728 
2 0.526614 
3 0.233053 
4 0.735908 
  

Title Description File Download SCGE ID
Chaikof-Associated Protocol 1_Retro-orbital administration and High throughput sequencing This Protocol describes in vivo administration of BE-eVLP for Pcsk9 knockdown in liver, including sequencing endpoint. Chaikof-Associated Protocol 1_Retro-orbital administration and High throughput sequencing.pdf 21000000031
Chaikof-Associated Protocol 3_Serum ELISAs This protocol describes in vivo adminstration and subsequent blood draws and ELISA on C57BL/6J mice. Chaikof-Associated Protocol 3_Serum ELISAs.pdf 21000000032

Publication Title
Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins. NCBI