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Validation Experiment: Independent validation of Chaikof delivery platform using virus-like particle (VLP) delivery to the mouse liver

PI:   Jason D Heaney, PhD

Description: Virus-like particles carrying a CRISPR/Cas base editor and a guide RNA targeting the PSCK9 locus were injected i.v. into male and female mice. One week after injection, the mice were dissected, and genomic DNA isolated from a panel of organs. Targeted NGS was performed to evaluate the degree of editing at the PCSK9 locus in the liver (primary target), and non-target organs. Two potential off-target editing sites (OT6 and OT7) were also sequenced.
Editing Assay: Targeted next generation sequencing (NGS)
Download: Submitted files
Original Experiment/s that are being validated:
Filters ..
  Tissues
 Heart
 Kidney
 Liver(TARGET)
 Lung
 Ovary
 Spleen
 Testis
  Qualifier
 OT6
 OT7
 PCSK9
Cell Types
 Unspecified
Editors
 ABE8e
Guide Target Locus
 Pcsk9 Exon 1 splice domain
Guides
 Pcsk9 Exon 1 splice domain
Delivery Systems
 eVLP
Models
 B6
  Sex
 Female
 Male
Select experimental variable to highlight records on the chart: 
 

Note: Hover over the bars to view additional information

Results
Independent_validation_of_Chaikof_delivery_platform_using_virus-like_particle_(VLP)_delivery_to_the_mouse_liver
Editing Efficiency
Record Id Condition Qualifier Tissue Cell Type Sex Age Editor Model Delivery Target Locus Guide Dosage Injection Frequency Editing Efficiency % of reads Image
15000003515 Male (Control) PCSK9 liver (TARGET) Male 6 weeks B6
15000003516 Male (Control) PCSK9 heart Male 6 weeks B6
15000003517 Male (Control) PCSK9 lung Male 6 weeks B6
15000003518 Male (Control) PCSK9 spleen Male 6 weeks B6
15000003519 Male (Control) PCSK9 kidney Male 6 weeks B6
15000003520 Male (Control) PCSK9 testis Male 6 weeks B6
15000003521 Male (Control) OT6 liver (TARGET) Male 6 weeks B6
15000003522 Male (Control) OT6 heart Male 6 weeks B6
15000003523 Male (Control) OT6 lung Male 6 weeks B6
15000003524 Male (Control) OT6 spleen Male 6 weeks B6
15000003525 Male (Control) OT6 kidney Male 6 weeks B6
15000003526 Male (Control) OT6 testis Male 6 weeks B6
15000003527 Male (Control) OT7 liver (TARGET) Male 6 weeks B6
15000003528 Male (Control) OT7 heart Male 6 weeks B6
15000003529 Male (Control) OT7 lung Male 6 weeks B6
15000003530 Male (Control) OT7 spleen Male 6 weeks B6
15000003531 Male (Control) OT7 kidney Male 6 weeks B6
15000003532 Male (Control) OT7 testis Male 6 weeks B6
15000003533 Female (Control) PCSK9 liver (TARGET) Female 6 weeks B6
15000003534 Female (Control) PCSK9 heart Female 6 weeks B6
15000003535 Female (Control) PCSK9 lung Female 6 weeks B6
15000003536 Female (Control) PCSK9 spleen Female 6 weeks B6
15000003537 Female (Control) PCSK9 kidney Female 6 weeks B6
15000003538 Female (Control) PCSK9 ovary Female 6 weeks B6
15000003539 Female (Control) OT6 liver (TARGET) Female 6 weeks B6
15000003540 Female (Control) OT6 heart Female 6 weeks B6
15000003541 Female (Control) OT6 lung Female 6 weeks B6
15000003542 Female (Control) OT6 spleen Female 6 weeks B6
15000003543 Female (Control) OT6 kidney Female 6 weeks B6
15000003544 Female (Control) OT6 ovary Female 6 weeks B6
15000003545 Female (Control) OT7 liver (TARGET) Female 6 weeks B6
15000003546 Female (Control) OT7 heart Female 6 weeks B6
15000003547 Female (Control) OT7 lung Female 6 weeks B6
15000003548 Female (Control) OT7 spleen Female 6 weeks B6
15000003549 Female (Control) OT7 kidney Female 6 weeks B6
15000003550 Female (Control) OT7 ovary Female 6 weeks B6
15000003551 Male (Experimental) PCSK9 liver (TARGET) Male 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003552 Male (Experimental) PCSK9 heart Male 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003553 Male (Experimental) PCSK9 lung Male 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003554 Male (Experimental) PCSK9 spleen Male 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003555 Male (Experimental) PCSK9 kidney Male 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003556 Male (Experimental) PCSK9 testis Male 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003557 Male (Experimental) OT6 liver (TARGET) Male 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003558 Male (Experimental) OT6 heart Male 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003559 Male (Experimental) OT6 lung Male 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003560 Male (Experimental) OT6 spleen Male 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003561 Male (Experimental) OT6 kidney Male 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003562 Male (Experimental) OT6 testis Male 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003563 Male (Experimental) OT7 liver (TARGET) Male 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003564 Male (Experimental) OT7 heart Male 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003565 Male (Experimental) OT7 lung Male 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003566 Male (Experimental) OT7 spleen Male 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003567 Male (Experimental) OT7 kidney Male 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003568 Male (Experimental) OT7 testis Male 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003569 Female (Experimental) PCSK9 liver (TARGET) Female 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003570 Female (Experimental) PCSK9 heart Female 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003571 Female (Experimental) PCSK9 lung Female 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003572 Female (Experimental) PCSK9 spleen Female 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003573 Female (Experimental) PCSK9 kidney Female 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003574 Female (Experimental) PCSK9 ovary Female 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003575 Female (Experimental) OT6 liver (TARGET) Female 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003576 Female (Experimental) OT6 heart Female 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003577 Female (Experimental) OT6 lung Female 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003578 Female (Experimental) OT6 spleen Female 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003579 Female (Experimental) OT6 kidney Female 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003580 Female (Experimental) OT6 ovary Female 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003581 Female (Experimental) OT7 liver (TARGET) Female 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003582 Female (Experimental) OT7 heart Female 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003583 Female (Experimental) OT7 lung Female 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003584 Female (Experimental) OT7 spleen Female 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003585 Female (Experimental) OT7 kidney Female 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once
15000003586 Female (Experimental) OT7 ovary Female 6 weeks ABE8e B6 eVLP Pcsk9 Exon 1 splice domain Pcsk9 Exon 1 splice domain 5E11 particles once