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Scooped by
BigField GEG Tech
July 17, 5:40 AM
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Cancer therapies called checkpoint inhibitor drugs, which ratchet up the immune system, work better when this bacterium is around.
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BigField GEG Tech
July 8, 6:23 AM
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Experimental treatment could offer a safer, cheaper alternative to CAR-T-cell therapies for disorders such as lupus.
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BigField GEG Tech
June 17, 7:04 AM
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After treatment with CAR-T cells - immune cells engineered to attack cancer - patients sometimes tell their doctors they feel like they have "brain fog," or forgetfulness and difficulty concentrating.
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Scooped by
BigField GEG Tech
June 5, 6:56 AM
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Investigators from Mass General Brigham and Beth Israel Deaconess Medical Center have developed STITCHR, a new gene editing tool that can insert therapeutic genes into specific locations without causing unwanted mutations.
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BigField GEG Tech
June 2, 11:16 AM
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BigField GEG Tech
May 27, 6:56 AM
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Imagine a super-charged immune cell that can launch a focused attack on stubborn solid tumors - a smart fighter that destroys cancer cells for days without tiring.
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BigField GEG Tech
May 20, 5:05 AM
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‘Directed’ evolution in the laboratory creates an editing tool that outperforms classic CRISPR systems.
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BigField GEG Tech
May 16, 6:25 AM
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diagnosis of severe carbamoyl-phosphate synthetase 1 deficiency
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BigField GEG Tech
April 24, 11:43 AM
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Congenital hearing loss refers to impaired auditory function that occurs due to genetic causes. GJB2 is the gene responsible for approximately half of all cases of hereditary hearing loss.
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BigField GEG Tech
April 3, 7:10 AM
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Chimeric antigen receptor (CAR) T cells and bispecific T cell engagers have become integral components in the treatment of relapsed/refractory multiple myeloma. We report a 63-year-old male who received ciltacabtagene autoleucel CAR T cells and the GPRC5D × CD3 bispecific talquetamab for early relapse of his multiple myeloma. Nine months after CAR T therapy, he developed a symptomatic leukemic peripheral T cell lymphoma with cutaneous and intestinal involvement. Longitudinal single-cell RNA and T cell receptor sequencing of peripheral blood and bone marrow revealed two hyperexpanded CAR-carrying T cell clones. These expanded clones exhibited an exhausted effector-memory T cell transcriptional signature, and the neoplasm itself was sensitive to dexamethasone treatment. The immunophenotypic and transcriptional alterations of these abnormal T cells resembled those of T-large granular lymphocytic leukemia. Spatial transcriptomes of skin lesions confirmed the aberrant CAR-expressing T cells. Whole-genome sequencing revealed three distinct integration sites, within the introns of ZGPAT, KPNA4 and polycomb-associated noncoding RNAs. Before and after CAR T whole-genome analyses implicated clonal outgrowth of a TET2-mutated precursor propelled by additional subclone-specific loss of heterozygosity and other secondary mechanisms. This case highlights the evolution of a CAR-carrying peripheral T cell lymphoma following CAR T cell and bispecific T cell engager therapy, offering critical insights into the clonal evolution from a predisposed hematopoietic precursor to a mature neoplasm. A longitudinal multiomics analysis of a patient with multiple myeloma who developed peripheral T cell lymphoma after treatment with anti-BCMA CAR T cells and a GPRC5D-directed bispecific antibody reveals that two mutated CAR+CD8+ T cell clones were probably drivers of the neoplasm.
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BigField GEG Tech
March 25, 3:22 AM
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Abstract. Human trisomy 21, responsible for Down syndrome, is the most prevalent genetic cause of cognitive impairment and remains a key focus for prenatal
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BigField GEG Tech
March 10, 7:11 AM
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Cas12a is a next-generation gene editing tool that enables multiplexed gene targeting. Here, we present a mouse model that constitutively expresses enhanced Acidaminococcus sp. Cas12a (enAsCas12a) linked to an mCherry fluorescent reporter. We demonstrate efficient single and multiplexed gene editing in vitro, using primary and transformed cells from enAsCas12a mice. We further demonstrate successful in vivo gene editing, using normal and cancer-prone enAsCas12a stem cells to reconstitute the haematopoietic system of wild-type mice. We also present compact, genome-wide Cas12a knockout libraries, with four crRNAs per gene encoded across one (Scherzo) or two (Menuetto) vectors, and demonstrate the utility of these libraries across methodologies: in vitro enrichment and drop-out screening in lymphoma cells and immortalised fibroblasts, respectively, and in vivo screens to identify lymphoma-driving events. Finally, we demonstrate CRISPR multiplexing via simultaneous gene knockout (via Cas12a) and activation (via dCas9-SAM) using primary T cells and fibroblasts. Our enAsCas12a mouse and accompanying crRNA libraries enhance genome engineering capabilities and complement current CRISPR technologies. Cas12a represents the next generation of gene editing. Here, the authors present the generation and validation of a Cas12a transgenic mouse model. Additionally, the authors create whole-genome Cas12a knockout libraries, and demonstrate their utility across multiple in vitro and in vivo screens.
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BigField GEG Tech
February 20, 12:00 PM
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CAR T cell therapy is one of the most promising new cancer treatments to emerge in recent years. It involves removing a patient's own immune T cells and engineering them to recognize specific targets on the surface of the cancer cell.
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Scooped by
BigField GEG Tech
July 10, 6:44 AM
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Chimeric Antigen Receptor (CAR)-T cells are a promising cancer therapy that are made from the patient's own T cells, which are reprogrammed to fight their cancer. One of the limitations of CAR-T cell therapy is the ability of these cells to survive long enough to target the entire tumor.
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BigField GEG Tech
July 7, 9:12 AM
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When a neuron in our body gets damaged, segments of RNA produce proteins that can help repair the injury.
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BigField GEG Tech
June 12, 6:29 AM
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Renier Brentjens, MD, PhD, discusses the current landscape of CAR T-cell agents in patients with solid tumors.
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BigField GEG Tech
June 4, 6:46 AM
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Trial in China is one of the first times the immune therapy has worked against solid tumours.
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BigField GEG Tech
June 2, 7:13 AM
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A new review outlines genetic engineering strategies, including CRISPR, to enhance the therapeutic potential of innate immune cells in cancer immunotherapy. The authors evaluate preclinical studies and emerging clinical data on how these cells can be retooled to overcome the limitations of conventional CAR T cell therapy.
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BigField GEG Tech
May 21, 10:22 AM
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Scooped by
BigField GEG Tech
May 19, 11:55 AM
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Genome editing has advanced at a rapid pace with promising results for treating genetic conditions-but there is always room for improvement.
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BigField GEG Tech
May 5, 9:01 AM
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Scientists deploy self-splicing protein subunits to insert strange new additions into target proteins.
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BigField GEG Tech
April 11, 5:57 AM
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Neurons engineered to evade the immune system could work as cell-replacement therapy.
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BigField GEG Tech
April 2, 11:20 AM
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A groundbreaking study in Nature Medicine reveals that GD2 CAR-T cell therapy can lead to long-term remission in children with neuroblastoma, with one patient remaining cancer-free for over 18 years.
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Scooped by
BigField GEG Tech
March 19, 7:00 AM
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The bioplastic was malleable, but is more expensive to produce than are plastics made from fossil fuels.
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Scooped by
BigField GEG Tech
March 4, 6:42 AM
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The programmable proteins are compact, modular, and can be directed to modify DNA in human cells.
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The CRISPR-Cas9 molecular complex is the tool most widely used by scientists around the world. It cuts double-stranded DNA at the exact point where the genetic material is to be modified. The cut activates two natural repair mechanisms, including homology-directed repair. To induce the cell to use this repair, researchers have recently begun using a molecule called AZD7648, which blocks rapid repair and forces the cell to use homology-directed repair. This approach should accelerate the development of more effective gene therapies. Initial studies on these new approaches have been positive. However, a research group has just discovered that the use of AZD7648 has serious side effects. The modifications would result in the outright deletion of thousands of DNA building blocks, known as bases. Entire chromosome arms were even detached, rendering the genome unstable, with unpredictable consequences for the cells modified by this technique. The study has just been published in the journal Nature Biotechnology.