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CRISPR-Cas9

New hPSC SOX9 and INS Reporter Cell Lines Facilitate the Observation and Optimization of Differentiation into Insulin-Producing Cells

[Stem Cell Reviews and Reports] For the purpose of easy monitoring of differentiation efficiencies into pancreatic progenitors and insulin-producing cells, scientists generated new reporter lines by knocking in a P2A-H-2Kk-F2A-GFP2 reporter gene into the SOX9-locus and a P2A-mCherry reporter gene into the INS-locus mediated by CRISPR/CAS9-technology.

Effective Control of Large Deletions after Double-Strand Breaks by Homology-Directed Repair and dsODN Insertion

[Genome Biology] The authors showed that large deletions were ubiquitous but were dependent on editing sites and cell types. Human primary T cells displayed more significant deletions than hematopoietic stem and progenitor cells, whereas they observed low levels in induced pluripotent stem cells.

CRISPR/Cas9 Correction of OPA1 c.1334G>A: p.R445H Restores Mitochondrial Homeostasis in Dominant Optic Atrophy Patient-Derived iPSCs

[Molecular Therapy-Nucleic Acids] Researchers demonstrated generation of patient-derived OPA1 c.1334G>A: p.R445H mutant iPSCs, followed by correction of OPA1 through CRISPR/Cas9 guided homology directed repair and evaluated the effect of OPA1 correction on mitochondrial homeostasis.

Intrinsic Activation of β-Catenin Signaling by CRISPR/Cas9-Mediated Exon Skipping Contributes to Immune Evasion in Hepatocellular Carcinoma

[Scientific Reports] Researchers established human and mouse hepatocellular carcinoma cells with exon 3 skipping of β-catenin, which promoted nuclear translocation and activated the Wnt/β-catenin signaling pathway, by using newly developed multiplex CRISPR/Cas9-based genome engineering system.

Cas9-Derived Peptides Presented by MHC Class II That Elicit Proliferation of CD4+ T-cells

[Nature Communications] Investigators used an MHC Associated Peptide Proteomics assay to identify SaCas9 peptides presented by MHC Class II (MHC-II) proteins on dendritic cells.

Targeted T Cell Receptor Gene Editing Provides Predictable T Cell Product Function for Immunotherapy

[Cell Reports Medicine] By studying 51 different T cell receptors (TCRs), scientists showed that conventional genetic engineering by viral transduction led to variable TCR expression and functionality as a result of variable transgene copy numbers and untargeted transgene integration.

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