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

Results

Independent_validation_of_Chaikof_delivery_platform_using_virus-like_particle_(VLP)_delivery_to_the_mouse_liver
											Editing Efficiency
Record Id	Condition	Qualifier	Tissue	Sex	Editor	Model	Delivery	Target Locus	Guide	Dosage	Injection Frequency	Editing Efficiency % of reads	Image
15000003515	Male (Control)	PCSK9	liver (TARGET)	Male		B6						0.1; 0.1;
15000003516	Male (Control)	PCSK9	heart	Male		B6						0.0; 0.0;
15000003517	Male (Control)	PCSK9	lung	Male		B6						0.0; 0.0;
15000003518	Male (Control)	PCSK9	spleen	Male		B6						0.0; 0.0;
15000003519	Male (Control)	PCSK9	kidney	Male		B6						0.0; 0.0;
15000003520	Male (Control)	PCSK9	testis	Male		B6						0.0; 0.0;
15000003521	Male (Control)	OT6	liver (TARGET)	Male		B6						0.0; 0.0;
15000003522	Male (Control)	OT6	heart	Male		B6						0.0; 0.0;
15000003523	Male (Control)	OT6	lung	Male		B6						0.0; 0.0;
15000003524	Male (Control)	OT6	spleen	Male		B6						0.0; 0.0;
15000003525	Male (Control)	OT6	kidney	Male		B6						0.0; 0.0;
15000003526	Male (Control)	OT6	testis	Male		B6						0.0; 0.0;
15000003527	Male (Control)	OT7	liver (TARGET)	Male		B6						0.0; 0.0;
15000003528	Male (Control)	OT7	heart	Male		B6						0.0; 0.0;
15000003529	Male (Control)	OT7	lung	Male		B6						0.0; 0.0;
15000003530	Male (Control)	OT7	spleen	Male		B6						0.0; 0.0;
15000003531	Male (Control)	OT7	kidney	Male		B6						0.0; 0.0;
15000003532	Male (Control)	OT7	testis	Male		B6						0.0; 0.0;
15000003533	Female (Control)	PCSK9	liver (TARGET)	Female		B6						0.0; 0.0;
15000003534	Female (Control)	PCSK9	heart	Female		B6						0.0; 0.0;
15000003535	Female (Control)	PCSK9	lung	Female		B6						0.0; 0.0;
15000003536	Female (Control)	PCSK9	spleen	Female		B6						0.0; 0.0;
15000003537	Female (Control)	PCSK9	kidney	Female		B6						0.0; 0.0;
15000003538	Female (Control)	PCSK9	ovary	Female		B6						0.0; 0.0;
15000003539	Female (Control)	OT6	liver (TARGET)	Female		B6						0.0; 0.0;
15000003540	Female (Control)	OT6	heart	Female		B6						0.0; 0.0;
15000003541	Female (Control)	OT6	lung	Female		B6						0.0; 0.0;
15000003542	Female (Control)	OT6	spleen	Female		B6						0.0; 0.0;
15000003543	Female (Control)	OT6	kidney	Female		B6						0.0; 0.0;
15000003544	Female (Control)	OT6	ovary	Female		B6						0.0; 0.0;
15000003545	Female (Control)	OT7	liver (TARGET)	Female		B6						0.0; 0.0;
15000003546	Female (Control)	OT7	heart	Female		B6						0.0; 0.0;
15000003547	Female (Control)	OT7	lung	Female		B6						0.0; 0.0;
15000003548	Female (Control)	OT7	spleen	Female		B6						0.0; 0.0;
15000003549	Female (Control)	OT7	kidney	Female		B6						0.0; 0.0;
15000003550	Female (Control)	OT7	ovary	Female		B6						0.0; 0.0;
15000003551	Male (Experimental)	PCSK9	liver (TARGET)	Male	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	32.8; 28.8; 41.9;
15000003552	Male (Experimental)	PCSK9	heart	Male	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.1; 0.1; 0.1;
15000003553	Male (Experimental)	PCSK9	lung	Male	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.1; 0.1;
15000003554	Male (Experimental)	PCSK9	spleen	Male	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	2.1; 2.9; 3.8;
15000003555	Male (Experimental)	PCSK9	kidney	Male	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.1; 0.1; 0.1;
15000003556	Male (Experimental)	PCSK9	testis	Male	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.1; 0.1;
15000003557	Male (Experimental)	OT6	liver (TARGET)	Male	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003558	Male (Experimental)	OT6	heart	Male	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003559	Male (Experimental)	OT6	lung	Male	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003560	Male (Experimental)	OT6	spleen	Male	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003561	Male (Experimental)	OT6	kidney	Male	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003562	Male (Experimental)	OT6	testis	Male	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003563	Male (Experimental)	OT7	liver (TARGET)	Male	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003564	Male (Experimental)	OT7	heart	Male	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003565	Male (Experimental)	OT7	lung	Male	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003566	Male (Experimental)	OT7	spleen	Male	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003567	Male (Experimental)	OT7	kidney	Male	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003568	Male (Experimental)	OT7	testis	Male	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003569	Female (Experimental)	PCSK9	liver (TARGET)	Female	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	56.5; 48.4; 59.6;
15000003570	Female (Experimental)	PCSK9	heart	Female	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.2; 0.1; 0.1;
15000003571	Female (Experimental)	PCSK9	lung	Female	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.2; 0.0; 0.1;
15000003572	Female (Experimental)	PCSK9	spleen	Female	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	3.4; 4.5; 4.6;
15000003573	Female (Experimental)	PCSK9	kidney	Female	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.1; 0.3; 0.3;
15000003574	Female (Experimental)	PCSK9	ovary	Female	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.2; 0.1; 0.3;
15000003575	Female (Experimental)	OT6	liver (TARGET)	Female	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003576	Female (Experimental)	OT6	heart	Female	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003577	Female (Experimental)	OT6	lung	Female	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003578	Female (Experimental)	OT6	spleen	Female	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003579	Female (Experimental)	OT6	kidney	Female	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003580	Female (Experimental)	OT6	ovary	Female	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003581	Female (Experimental)	OT7	liver (TARGET)	Female	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003582	Female (Experimental)	OT7	heart	Female	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003583	Female (Experimental)	OT7	lung	Female	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003584	Female (Experimental)	OT7	spleen	Female	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003585	Female (Experimental)	OT7	kidney	Female	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;
15000003586	Female (Experimental)	OT7	ovary	Female	ABE8e	B6	eVLP	Pcsk9 Exon 1 splice domain	Pcsk9 Exon 1 splice domain	5E11 particles	once	0.0; 0.0; 0.0;

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

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

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Results