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Double step screening using endogenous marker improves relative gene targeting efficiency in Arabidopsis.
Sci Rep
December 2024
Shanghai Center for Plant Stress Biology, CAS Center for Excellence in Molecular Plant Sciences, Chinese Academy of Sciences, Shanghai, 200032, China.
Gene targeting (GT) is a powerful tool for manipulating endogenous genomic sequences as intended. However, its efficiency is rather low, especially in seed plants. Numerous attempts have been made to improve the efficiency of GT via the CRISPR/Cas systems in plants, but these have not been sufficiently effective to be used routinely by everyone.
View Article and Find Full Text PDFTonabersat enhances temozolomide-mediated cytotoxicity in glioblastoma by disrupting intercellular connectivity through connexin 43 inhibition.
Mol Oncol
December 2024
Department of Neurosurgery, University Hospital Bonn, Germany.
Glioblastoma cells rely on connexin 43 (Cx43)-based gap junctions (GJs) for intercellular communication, enabling them to integrate into a widely branched malignant network. Although there are promising prospects for new targeted therapies, the lack of clinically feasible GJ inhibitors has impeded their adoption in clinical practice. In the present study, we investigated tonabersat (TO), a blood-brain-barrier-penetrating drug with GJ-inhibitory properties, in regard to its potential to disassemble intercellular connectivity in glioblastoma networks.
View Article and Find Full Text PDFRestored Collagen VI Microfilaments Network in the Extracellular Matrix of CRISPR-Edited Ullrich Congenital Muscular Dystrophy Fibroblasts.
Biomolecules
November 2024
Department of Life Sciences, University of Modena and Reggio Emilia, 41125 Modena, Italy.
Article Synopsis
- Collagen VI, made of three chains encoded by specific genes, is crucial for the extracellular matrix and its defects are linked to muscular diseases like Ullrich congenital muscular dystrophy (UCMD).
- A CRISPR genome editing strategy was developed to specifically target a harmful deletion in the collagen VI gene, achieving 32% editing efficiency and restoring the protein's secretion in patient fibroblasts.
- The edited fibroblasts not only improved collagen VI levels but also positively impacted cell interactions, suggesting that CRISPR can effectively address genetic mutations in UCMD.
Blastocyst complementation generates exogenous donor-derived liver in ahepatic pigs.
FASEB J
November 2024
RenOVAte Biosciences Inc, Reisterstown, Maryland, USA.
Liver diseases are one of the leading causes of morbidity and mortality worldwide. Globally, liver diseases are responsible for approximately 2 million deaths annually (1 of every 25 deaths). Many of the patients with chronic liver diseases can benefit from organ transplantation.
View Article and Find Full Text PDFMicroglia replacement by ER-Hoxb8 conditionally immortalized macrophages provides insight into Aicardi-Goutières Syndrome neuropathology.
bioRxiv
September 2024
Department of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Microglia, the brain's resident macrophages, can be reconstituted by surrogate cells - a process termed "microglia replacement." To expand the microglia replacement toolkit, we here introduce estrogen-regulated (ER) homeobox B8 (Hoxb8) conditionally immortalized macrophages, a cell model for generation of immune cells from murine bone marrow, as a versatile model for microglia replacement. We find that ER-Hoxb8 macrophages are highly comparable to primary bone marrow-derived (BMD) macrophages in vitro, and, when transplanted into a microglia-free brain, engraft the parenchyma and differentiate into microglia-like cells.
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