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)
Parent Project: Delivery Technologies for In Vivo Genome Editing
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Other experiments in this project: 1
Download: Submitted files
Publications:
- Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins. NCBI
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Results |
| Editing Efficiency | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Condition | Tissue | Cell Type | Sex | Age | Editor | Model | Delivery | Target Locus | Guide | Injection Frequency | % of total sequencing reads with AT converted to GC | Image |
| BE-eVLP-Pcsk9 | heart | Male | 6-8 weeks | TadA-8e V106W | C57BL/6J mouse | eVLP | Pcsk9 Exon 1 splice domain | Pcsk9 Exon 1 splice domain | once | |||
| BE-eVLP-Pcsk9 | kidney | Male | 6-8 weeks | TadA-8e V106W | C57BL/6J mouse | eVLP | Pcsk9 Exon 1 splice domain | Pcsk9 Exon 1 splice domain | once | |||
| BE-eVLP-Pcsk9 | liver (TARGET) | Male | 6-8 weeks | TadA-8e V106W | C57BL/6J mouse | eVLP | Pcsk9 Exon 1 splice domain | Pcsk9 Exon 1 splice domain | once | |||
| BE-eVLP-Pcsk9 | lung | Male | 6-8 weeks | TadA-8e V106W | C57BL/6J mouse | eVLP | Pcsk9 Exon 1 splice domain | Pcsk9 Exon 1 splice domain | once | |||
| BE-eVLP-Pcsk9 | muscle tissue | Male | 6-8 weeks | TadA-8e V106W | C57BL/6J mouse | eVLP | Pcsk9 Exon 1 splice domain | Pcsk9 Exon 1 splice domain | once | |||
| BE-eVLP-Pcsk9 | spleen | Male | 6-8 weeks | TadA-8e V106W | C57BL/6J mouse | eVLP | Pcsk9 Exon 1 splice domain | Pcsk9 Exon 1 splice domain | once | |||
| Untreated | heart | Male | 6-8 weeks | C57BL/6J mouse | ||||||||
| Untreated | kidney | Male | 6-8 weeks | C57BL/6J mouse | ||||||||
| Untreated | liver (TARGET) | Male | 6-8 weeks | C57BL/6J mouse | ||||||||
| Untreated | lung | Male | 6-8 weeks | C57BL/6J mouse | ||||||||
| Untreated | muscle tissue | Male | 6-8 weeks | C57BL/6J mouse | ||||||||
| Untreated | spleen | Male | 6-8 weeks | 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). |
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