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Experiment: Pcsk9 adenine base editor efficiency in liver and nonliver tissue

PI:   Elliot L. Chaikof, MD, PhD 

Description: Adenine base editing at the Pcsk9 exon 1 splice donor site in mouse heart, kidney, liver, lungs, muscle, and spleen was assessed one week after systemic administration of an adenine base editor delivered by engineered virus-like particles (BE-eVLPs) in C56BL/6 mice
Editing Assay: adenine base editing at target Pcsk9 site in liver vs. other mouse organs (heart, kidney, lung, muscle, spleen)
Download: Submitted files
Publications: 
  • Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins. NCBI
Filters ..
  Tissues
Renal/Urinary
  • Kidney
Haemolymphoid
  • Spleen
Liver and Biliary
  • Liver  (TARGET)
Respiratory
  • Lung
Cardiovascular
  • Heart
Musculoskeletal
  • Muscle tissue
Editors
 TadA-8e V106W
Guide Target Locus
 Pcsk9 Exon 1 splice domain
Guides
 Pcsk9 Exon 1 splice domain
Delivery Systems
 eVLP
Models
 C57BL/6J mouse
Sex
 Male
Select experimental variable to highlight records on the chart: 
 

Note: Hover over the bars to view additional information

Results
Pcsk9_adenine_base_editor_efficiency_in_liver_and_nonliver_tissue
Editing Efficiency
Record Id Condition Tissue Sex Editor Model Delivery Target Locus Guide Injection Frequency Editing Efficiency % of total sequencing reads with AT converted to GC Image
15000001520 BE-eVLP-Pcsk9 heart Male TadA-8e V106W C57BL/6J mouse eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain once
15000001521 BE-eVLP-Pcsk9 kidney Male TadA-8e V106W C57BL/6J mouse eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain once
15000001522 BE-eVLP-Pcsk9 liver (TARGET) Male TadA-8e V106W C57BL/6J mouse eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain once
15000001523 BE-eVLP-Pcsk9 lung Male TadA-8e V106W C57BL/6J mouse eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain once
15000001524 BE-eVLP-Pcsk9 muscle tissue Male TadA-8e V106W C57BL/6J mouse eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain once
15000001525 BE-eVLP-Pcsk9 spleen Male TadA-8e V106W C57BL/6J mouse eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain once
15000001526 Untreated heart Male C57BL/6J mouse
15000001527 Untreated kidney Male C57BL/6J mouse
15000001528 Untreated liver (TARGET) Male C57BL/6J mouse
15000001529 Untreated lung Male C57BL/6J mouse
15000001530 Untreated muscle tissue Male C57BL/6J mouse
15000001531 Untreated spleen Male C57BL/6J mouse

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

Experiment Wide Protocols

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
Adenine base editing efficiency in liver versus nonliver tissue

Adenine base editing efficiencies at the Pcsk9 exon 1 splice donor in the mouse heart, kidney, liver, lungs, muscle, and spleen after systemic injection of 7 x 10E11 BE-eVLPs. Data are shown as individual data points and mean +/- SEM for n=4 mice (treated) or n=3 mice (untreated)
Serum Pcsk9 levels in untreated versus BE-eVLP knockdown mice

Serum Pcsk9 levels as measured by ELISA. Data are shown as individual data points and mean +/- SEM for n=4 mice (treated) or n=3 mice (untreated).