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248 results for “Gene editing”
Sanger sequencing of target and off-target genomic regions for gene-edited iPSC clones with SETBP1 genetic variants
<p>This data set includes chromatograms generated using sanger sequencing of targeted regions of genomic DNA from clonal iPSC lines. The iPSC lines include clones generated using CRISPR/Cas9 homology directed repair to introduce genetic variants into <em>SETBP1,</em> and their wild-type controls. Additional files have been included in the data set to link chromatogram (ab1) files to specific iPSC clones for genomic regions across the variant in <em>SETBP1 (</em>SETBP1 clones genetic variant sanger sequencing.xslx)<em> </em>and top<em> </em>off-target sites (SETBP1 clones off-target sanger sequencing.xlsx). </p>
Data from "GEARBOCS: An Adeno Associated Virus Tool for In Vivo Gene Editing in Astrocytes"
<p>Data from "GEARBOCS: An Adeno Associated Virus Tool for In Vivo Gene Editing in Astrocytes". Folders contain data corresponding to each figure. Additional readme files are included with each figure folder to explain the data contained there.</p>
Safety and Efficacy of CRISPR/Cas9 mRNA Instantaneous Gene Editing Therapy to Treat Refractory Viral Keratitis
ClinicalTrials.gov study NCT04560790. IPD Sharing: NO. Countries: 1. Publications: 1.
A Study of Gene Edited Autologous Neoantigen Targeted TCR T Cells With or Without Anti-PD-1 in Patients With Solid Tumors
ClinicalTrials.gov study NCT03970382. IPD Sharing: NO. Countries: 1. Publications: 2.
Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study
ClinicalTrials.gov study NCT06959771. IPD Sharing: UNDECIDED. Countries: 1. Publications: 8.
Data from: Chromosome-level reference genome assembly and gene editing of the dead-leaf butterfly Kallima inachus
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The amount of RNA editing sites in liverwort organellar genes is correlated with GC content and nuclear PPR protein diversity
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Data from: Gene editing to induce FOXP3 expression in human CD4+ T cells leads to a stable regulatory phenotype and function.
<p>Thymic regulatory T cells (tTreg) are potent inhibitors of autoreactive immune responses and loss of tTreg function results in fatal autoimmune disease. Defects in Treg number or function are also implicated in multiple autoimmune diseases, leading to growing interest in use of Treg as cell therapies to establish immune tolerance. Because tTreg are present at low numbers in circulating blood and may be challenging to purify and expand, and also inherently defective in some subjects, we designed an alternative strategy to creating autologous Treg-like cells from bulk CD4+ T cells. We utilized homology-directed-repair (HDR)-based gene-editing to enforce FOXP3 expression. Targeted insertion of a robust enhancer/promoter proximal to the first coding exon bypassed epigenetic silencing, permitting stable, high level endogenous FOXP3 expression. HDR-edited T cells, edTreg, manifested a transcriptional program leading to sustained expression of canonical markers and suppressive activity of tTreg. Both human and murine edTreg mediated immunosuppression in vivo in models of inflammatory disease. Further, this engineering strategy permitted generation of antigen-specific edTreg with robust in vitro and in vivo functional activity. Finally, edTreg could be enriched and expanded at scale using clinically-relevant methods. Together, these finding suggest edTreg production may permit broad future clinical application.</p>
Source data for "Optimizing 5'UTRs for mRNA-delivered gene editing using deep learning"
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Gene Editing For Sickle Cell Disease
ClinicalTrials.gov study NCT06506461. IPD Sharing: YES. Countries: 1. Publications: 0.
Data from: Gene editing to induce FOXP3 expression in human CD4+ T cells leads to a stable regulatory phenotype and function.
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Genomic profile of gene edited hematopoietic stem cells
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TALENs-mediated gene disruption of FLT3 in leukemia cells: Using genome-editing approach for exploring the molecular basis of gene abnormality
GEO Series GSE69678. Homo sapiens. 6 samples. Type: Expression profiling by high throughput sequencing.
Durable and efficient gene silencing in vivo by hit-and-run epigenome editing [deep_sequencing_ZFPoff8]
GEO Series GSE240937. Mus musculus. 6 samples. Type: Other.
Joint single-cell profiling of Cas9 edits and transcriptomes reveals on- and off-target effects on gene expression (RNA-seq)
GEO Series GSE313958. Homo sapiens. 6 samples. Type: Expression profiling by high throughput sequencing.
Single cell transcriptomic profiling of day 30 patient-specific pluripotent stem cell-derived alveolar epithelial type 2 cells expressing the SFTPCI73T mutation and their gene-edited corrected counter
GEO Series GSE160798. Homo sapiens. 2 samples. Type: Expression profiling by high throughput sequencing.
Lipid Nanoparticles Allow Efficient and Harmless Ex Vivo Gene Editing of Human Hematopoietic Cells [RNAseq_lnps]
GEO Series GSE216249. Homo sapiens. 36 samples. Type: Expression profiling by high throughput sequencing.
Expanding the editable genome and CRISPR-Cas9 versatility using DNA cutting-free gene targeting based on in trans paired nicking
GEO Series GSE135064. Homo sapiens. 12 samples. Type: Other.
ADAR2 affects mRNA coding sequence edits but not gene expression or splicing in vivo
GEO Series GSE70588. Mus musculus. 12 samples. Type: Expression profiling by high throughput sequencing.
Linking CRISPR/Cas9 double-strand break profiles to gene editing precision with BreakTag [hiplex3]
GEO Series GSE223769. Homo sapiens. 30 samples. Type: Other.
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Allen Brain Atlas
Allen Brain Atlas is an Allen Institute collection of brain map atlases, datasets, APIs, and analysis tools covering mouse, human, and non-human primate brain resources.
Annotated Behaviour and Observability Dataset (ABODe)
ABODe is a University of Edinburgh DataShare dataset for behavior classification in group-housed mice using home-cage video, identities, bounding boxes, ground-plate positions, and annotator labels.
DANDI Archive for NWB datasets
DANDI is a BRAIN Initiative archive for publishing and sharing neurophysiology data, including electrophysiology, optophysiology, and behavioral data packaged as NWB and related standards.
International Brain Laboratory public data
The International Brain Laboratory public data releases expose standardized mouse decision-making experiments, including Neuropixels recordings, widefield calcium imaging, behavior, and session metadata accessed through the ONE API.
OpenNeuro
OpenNeuro is a free, open platform for sharing neuroimaging datasets, with public search, dataset pages, and download paths for web, S3, DataLad, and the OpenNeuro CLI.