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CRISPR-Cas9

NaV1.5 Knockout in iPSCs: A Novel Approach to Study NaV1.5 Variants in a Human Cardiomyocyte Environment

[Biophysical Journal] Scientists developed a homozygous NaV1.5 KO iPSC line able to differentiate into cardiomyocytes (iPSC-CMs) with CRISPR/Cas9 tool. The NaV1.5 KO iPSC-CMs exhibited an organized contractile apparatus, spontaneous contractile activity, and electrophysiological recordings confirmed the major reduction in total Na+ currents.

DGKZ Promotes TGFβ Signaling Pathway and Metastasis in Triple-Negative Breast Cancer by Suppressing Lipid Raft-Dependent Endocytosis of TGFβR2

[Cell Death & Disease] Knockout of diacylglycerol kinase ζ (DGKZ) in TNBC cell lines were found to significantly inhibit metastatic behaviors in vitro and in vivo, whereas its overexpression increased the metastatic potential of cell lines.

NPRL3 Loss Alters Neuronal Morphology, mTOR Localization, Cortical Lamination, and Seizure Threshold

[Brain] To model human malformations of cortical development associated with nitrogen permease regulator-like 3 (NPRL3) variants, researchers created a focal Nprl3 KO in fetal mouse cortex by in utero electroporation and found altered cortical lamination and white matter heterotopic neurons

CD38 Knockout Natural Killer Cells Expressing an Affinity Optimized CD38 Chimeric Antigen Receptor Successfully Target Acute Myeloid Leukemia with Reduced Effector Cell Fratricide

[Haematologica] Researchers developed a CD38 CAR-NK cell therapy for acute myeloid leukemia, first by using an NK cell line which had low baseline CD38 expression and subsequently NK cells expanded from healthy donors.

Human iPSC Model Reveals a Central Role for NOX4 and Oxidative Stress in Duchenne Cardiomyopathy

[Stem Cell Reports] The authors used Duchenne muscular dystrophy-patient-specific human iPSCs to model cardiomyopathic features and unravel novel pathologic insights.

An In Vivo Cell-Based Delivery Platform for Zinc Finger Artificial Transcription Factors in Pre-Clinical Animal Models

[Frontiers in Molecular Neuroscience] Researchers described the use of an MSC-based delivery system for the secretion of a Zinc fingers protein in transgenic mouse models and young rhesus monkeys.

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