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cdna  (Addgene inc)


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    Addgene inc cdna
    Figure 1. Targeted integration of WAS2-12 sequences into WAS intron 1 of normal male CD34+ cells (A) Schematic of WAS intron 1 targeted integration (TI) strategy. Shown is the location of CRISPR-Cas9 DNA cleavage in the endogenous WAS intron 1 (top diagram), the AAV6 delivered donor template (middle diagram), and the desired edited locus (bottom diagram). (B) Percentage of WAS intron 1 sequences that were modified after electroporation of CD34+ HSPCs with Cas9/gRNA RNP. (C) Quantification of on-target (ON) and off-target (OT) activity by NGS and CRISPResso2 analysis of the ON and OT locations identified by COSMID (OT1-OT33) and GUIDE-seq (OT34-OT44). (D) AAV-6 donor vectors employed in this study including therapeutic donors containing WAS2-12 <t>cDNA,</t> a splice acceptor (SA), poly(A) sequence <t>(pA),</t> <t>2A</t> sequence (2A), phosphoglycerate kinase (PGK) promoter, and a GFP reporter gene (GFP). (E) Experimental timeline for editing of primary CD34+
    Cdna, supplied by Addgene inc, used in various techniques. Bioz Stars score: 93/100, based on 18 PubMed citations. ZERO BIAS - scores, article reviews, protocol conditions and more
    https://www.bioz.com/result/cdna/product/Addgene inc
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    Images

    1) Product Images from "Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome."

    Article Title: Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome.

    Journal: Molecular therapy. Methods & clinical development

    doi: 10.1016/j.omtm.2024.101208

    Figure 1. Targeted integration of WAS2-12 sequences into WAS intron 1 of normal male CD34+ cells (A) Schematic of WAS intron 1 targeted integration (TI) strategy. Shown is the location of CRISPR-Cas9 DNA cleavage in the endogenous WAS intron 1 (top diagram), the AAV6 delivered donor template (middle diagram), and the desired edited locus (bottom diagram). (B) Percentage of WAS intron 1 sequences that were modified after electroporation of CD34+ HSPCs with Cas9/gRNA RNP. (C) Quantification of on-target (ON) and off-target (OT) activity by NGS and CRISPResso2 analysis of the ON and OT locations identified by COSMID (OT1-OT33) and GUIDE-seq (OT34-OT44). (D) AAV-6 donor vectors employed in this study including therapeutic donors containing WAS2-12 cDNA, a splice acceptor (SA), poly(A) sequence (pA), 2A sequence (2A), phosphoglycerate kinase (PGK) promoter, and a GFP reporter gene (GFP). (E) Experimental timeline for editing of primary CD34+
    Figure Legend Snippet: Figure 1. Targeted integration of WAS2-12 sequences into WAS intron 1 of normal male CD34+ cells (A) Schematic of WAS intron 1 targeted integration (TI) strategy. Shown is the location of CRISPR-Cas9 DNA cleavage in the endogenous WAS intron 1 (top diagram), the AAV6 delivered donor template (middle diagram), and the desired edited locus (bottom diagram). (B) Percentage of WAS intron 1 sequences that were modified after electroporation of CD34+ HSPCs with Cas9/gRNA RNP. (C) Quantification of on-target (ON) and off-target (OT) activity by NGS and CRISPResso2 analysis of the ON and OT locations identified by COSMID (OT1-OT33) and GUIDE-seq (OT34-OT44). (D) AAV-6 donor vectors employed in this study including therapeutic donors containing WAS2-12 cDNA, a splice acceptor (SA), poly(A) sequence (pA), 2A sequence (2A), phosphoglycerate kinase (PGK) promoter, and a GFP reporter gene (GFP). (E) Experimental timeline for editing of primary CD34+

    Techniques Used: CRISPR, Electroporation, Activity Assay, Sequencing



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    Figure 1. Targeted integration of WAS2-12 sequences into WAS intron 1 of normal male CD34+ cells (A) Schematic of WAS intron 1 targeted integration (TI) strategy. Shown is the location of CRISPR-Cas9 DNA cleavage in the endogenous WAS intron 1 (top diagram), the AAV6 delivered donor template (middle diagram), and the desired edited locus (bottom diagram). (B) Percentage of WAS intron 1 sequences that were modified after electroporation of CD34+ HSPCs with Cas9/gRNA RNP. (C) Quantification of on-target (ON) and off-target (OT) activity by NGS and CRISPResso2 analysis of the ON and OT locations identified by COSMID (OT1-OT33) and GUIDE-seq (OT34-OT44). (D) AAV-6 donor vectors employed in this study including therapeutic donors containing WAS2-12 <t>cDNA,</t> a splice acceptor (SA), poly(A) sequence <t>(pA),</t> <t>2A</t> sequence (2A), phosphoglycerate kinase (PGK) promoter, and a GFP reporter gene (GFP). (E) Experimental timeline for editing of primary CD34+
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    Image Search Results


    Figure 1. Targeted integration of WAS2-12 sequences into WAS intron 1 of normal male CD34+ cells (A) Schematic of WAS intron 1 targeted integration (TI) strategy. Shown is the location of CRISPR-Cas9 DNA cleavage in the endogenous WAS intron 1 (top diagram), the AAV6 delivered donor template (middle diagram), and the desired edited locus (bottom diagram). (B) Percentage of WAS intron 1 sequences that were modified after electroporation of CD34+ HSPCs with Cas9/gRNA RNP. (C) Quantification of on-target (ON) and off-target (OT) activity by NGS and CRISPResso2 analysis of the ON and OT locations identified by COSMID (OT1-OT33) and GUIDE-seq (OT34-OT44). (D) AAV-6 donor vectors employed in this study including therapeutic donors containing WAS2-12 cDNA, a splice acceptor (SA), poly(A) sequence (pA), 2A sequence (2A), phosphoglycerate kinase (PGK) promoter, and a GFP reporter gene (GFP). (E) Experimental timeline for editing of primary CD34+

    Journal: Molecular therapy. Methods & clinical development

    Article Title: Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome.

    doi: 10.1016/j.omtm.2024.101208

    Figure Lengend Snippet: Figure 1. Targeted integration of WAS2-12 sequences into WAS intron 1 of normal male CD34+ cells (A) Schematic of WAS intron 1 targeted integration (TI) strategy. Shown is the location of CRISPR-Cas9 DNA cleavage in the endogenous WAS intron 1 (top diagram), the AAV6 delivered donor template (middle diagram), and the desired edited locus (bottom diagram). (B) Percentage of WAS intron 1 sequences that were modified after electroporation of CD34+ HSPCs with Cas9/gRNA RNP. (C) Quantification of on-target (ON) and off-target (OT) activity by NGS and CRISPResso2 analysis of the ON and OT locations identified by COSMID (OT1-OT33) and GUIDE-seq (OT34-OT44). (D) AAV-6 donor vectors employed in this study including therapeutic donors containing WAS2-12 cDNA, a splice acceptor (SA), poly(A) sequence (pA), 2A sequence (2A), phosphoglycerate kinase (PGK) promoter, and a GFP reporter gene (GFP). (E) Experimental timeline for editing of primary CD34+

    Article Snippet: Rawlings, Addgene, no. 36250) and the WAS1-12-2A-GFP expression cassette contains the WAS cDNA (exons 1–12) fused to eGFP via 2A sequences.

    Techniques: CRISPR, Electroporation, Activity Assay, Sequencing

    Fig. 4 | C5 locks SLC15A4 in an outward-open, TASL binding-incompetent conformation. a, b Ribbon model of human SLC15A4/C5 complex. In a the two protomers of SLC15A4 are colored pink and blue, respectively. The bound C5 molecules are shown assticks and colored dark yellow. In b the TMs surrounding C5 are highlighted. c, d Heatmap of the surface electrostatics of human SLC15A4 in the outward-open C5-bound state showing the C5 binding pocket. The C5 molecule is

    Journal: Nature communications

    Article Title: A conformation-locking inhibitor of SLC15A4 with TASL proteostatic anti-inflammatory activity.

    doi: 10.1038/s41467-023-42070-3

    Figure Lengend Snippet: Fig. 4 | C5 locks SLC15A4 in an outward-open, TASL binding-incompetent conformation. a, b Ribbon model of human SLC15A4/C5 complex. In a the two protomers of SLC15A4 are colored pink and blue, respectively. The bound C5 molecules are shown assticks and colored dark yellow. In b the TMs surrounding C5 are highlighted. c, d Heatmap of the surface electrostatics of human SLC15A4 in the outward-open C5-bound state showing the C5 binding pocket. The C5 molecule is

    Article Snippet: Fusions of TASL N-terminal sequences (aa 1-12, 1-13, 1-14 and 1-15) with GFP were generated by introducing corresponding annealed oligos into the BsmBI sites of the Artichoke reporter vector (Addgene Plasmid # 73320, kind gift from Benjamin Ebert) via Golden Gate assembly.

    Techniques: Binding Assay