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BigField GEG Tech
February 6, 5:58 AM
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This study is led by Prof. Xianqun Fan (Department of Ophthalmology, Shanghai Jiao Tong University School of Medicine Affiliated Ninth People's Hospital).
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BigField GEG Tech
February 2, 6:13 AM
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Chimeric antigen receptor T-cell therapy, or CAR T, has dramatically improved the treatment of certain blood cancers. Initially approved for patients who had failed multiple lines of therapy, clinical trials have shown CAR T can be used as an earlier treatment option.
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BigField GEG Tech
January 31, 9:10 AM
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BigField GEG Tech
January 16, 6:27 AM
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At EPFL's School of Engineering, Professor Li Tang's Laboratory of Biomaterials for Immunoengineering has made significant strides in cancer treatment research.
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BigField GEG Tech
December 21, 2023 6:14 AM
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The recent publication in Science by Mogila, Tamulaitiene et al. represents a continuation of the successful scientific research conducted by Gintautas Tamulaitis' group.
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BigField GEG Tech
December 19, 2023 5:42 AM
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The gene-editing technology CRISPR shows early promise as a therapeutic strategy for the aggressive and difficult-to-treat brain cancer known as primary glioblastoma, according to findings of a new study from Gladstone Institutes.
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BigField GEG Tech
November 22, 2023 8:47 AM
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RAG2-SCID is a primary immunodeficiency caused by mutations in Recombination-activating gene 2 (RAG2), a gene intimately involved in the process of lymphocyte maturation and function. ex-vivo manipulation of a patient’s own hematopoietic stem and progenitor cells (HSPCs) using CRISPR-Cas9/rAAV6 gene editing could provide a therapeutic alternative to the only current treatment, allogeneic hematopoietic stem cell transplantation (HSCT). Here we show an innovative RAG2 correction strategy that replaces the entire endogenous coding sequence (CDS) for the purpose of preserving the critical endogenous spatiotemporal gene regulation and locus architecture. Expression of the corrective transgene leads to successful development into CD3+TCRαβ+ and CD3+TCRγδ+ T cells and promotes the establishment of highly diverse TRB and TRG repertoires in an in-vitro T-cell differentiation platform. Thus, our proof-of-concept study holds promise for safer gene therapy techniques of tightly regulated genes. RAG2-SCID is a primary immunodeficiency caused by mutations in Recombination-activating gene 2 (RAG2). Here the authors report a RAG2 correction strategy that replaces the entire endogenous coding sequence (CDS) to maintain the endogenous spatiotemporal gene regulation and locus architecture.
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BigField GEG Tech
September 22, 2023 6:29 AM
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Rochester Institute of Technology researchers are improving non-invasive treatment options for degenerative disc disease, an ailment that impacts 3 million adults yearly in the U.S., according to the Mayo Clinic.
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BigField GEG Tech
July 26, 2023 7:07 AM
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Scientists at Leipzig University, in collaboration with colleagues at Vilnius University in Lithuania, have developed a new method to measure the smallest twists and torques of molecules within milliseconds.
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BigField GEG Tech
July 24, 2023 10:57 AM
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Vaccine boosting modifies CAR T cell metabolism and promotes crosstalk between CAR
T cells and endogenous immunity to elicit and sustain antigen spreading, thereby effectively
treating tumors with antigen heterogeneity.
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BigField GEG Tech
July 19, 2023 7:19 AM
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Molecular routes to metastatic dissemination are critical determinants of aggressive cancers. Through in vivo CRISPR–Cas9 genome editing, we generated somatic mosaic genetically engineered models that faithfully recapitulate metastatic renal tumors. Disruption of 9p21 locus is an evolutionary driver to systemic disease through the rapid acquisition of complex karyotypes in cancer cells. Cross-species analysis revealed that recurrent patterns of copy number variations, including 21q loss and dysregulation of the interferon pathway, are major drivers of metastatic potential. In vitro and in vivo genomic engineering, leveraging loss-of-function studies, along with a model of partial trisomy of chromosome 21q, demonstrated a dosage-dependent effect of the interferon receptor genes cluster as an adaptive mechanism to deleterious chromosomal instability in metastatic progression. This work provides critical knowledge on drivers of renal cell carcinoma progression and defines the primary role of interferon signaling in constraining the propagation of aneuploid clones in cancer evolution. Using genetically engineered models, Genovese and colleagues study patterns of convergent evolution in renal cancer, and pinpoint dysregulation of interferon signaling as a means of adaptation to chromosomal instability in metastatic progression.
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BigField GEG Tech
July 4, 2023 9:57 AM
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Three young patients with relapsed T-cell leukemia have now been treated with base-edited T-cells, as part of a 'bench-to-bedside' collaboration between UCL and Great Ormond Street Hospital for Children (GOSH).
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BigField GEG Tech
June 15, 2023 6:29 AM
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The most common form of genetic heart disease is hypertrophic cardiomyopathy (HCM), which is caused by variants in cardiac sarcomeric genes and leads to abnormal heart muscle thickening. Complications of HCM include heart failure, arrhythmia and sudden cardiac death. The dominant-negative c.1208G>A (p.R403Q) pathogenic variant (PV) in β-myosin (MYH7) is a common and well-studied PV that leads to increased cardiac contractility and HCM onset. In this study we identify an adenine base editor and single-guide RNA system that can efficiently correct this human PV with minimal bystander editing and off-target editing at selected sites. We show that delivery of base editing components rescues pathological manifestations of HCM in induced pluripotent stem cell cardiomyocytes derived from patients with HCM and in a humanized mouse model of HCM. Our findings demonstrate the potential of base editing to treat inherited cardiac diseases and prompt the further development of adenine base editor-based therapies to correct monogenic variants causing cardiac disease. Adenine base editing successfully corrected a MYH7 pathogenic variant that causes hypertrophic cardiomyopathy in human cardiomyocytes and a mouse model of the disease, highlighting the potential of the approach to correct monogenic variants causing cardiac disease.
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BigField GEG Tech
February 5, 6:11 AM
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A new light-inducible RNA base editing tool, padCas13, combines the specificity of CRISPR-Cas13 with the control of light activation and allows for precise, reversible RNA targeting and degradation in mammalian cells, both in vitro and in vivo.
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BigField GEG Tech
February 1, 11:56 AM
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Using CRISPR, an immune system bacteria use to protect themselves from viruses, scientists have harnessed the power to edit genetic information within cells.
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BigField GEG Tech
January 17, 6:06 AM
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Researchers developed an RNA-based switch, the pA regulator system, to control gene expression in mammalian cells by modulating synthetic polyA signal cleavage, offering a novel approach for gene therapy applications.
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BigField GEG Tech
December 26, 2023 10:36 AM
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Utilizing CRISPR screening, the deubiquitinase ATXN3 has been identified as a key regulator of PD-L1 transcription in tumor cells, a critical factor in tumor immune evasion.
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BigField GEG Tech
December 20, 2023 9:00 AM
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BigField GEG Tech
December 18, 2023 6:31 AM
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Immunotherapy using modified chimeric antigen receptor (CAR) T cells has greatly improved survival rates for pediatric patients with relapsed and recurrent leukemia.
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In a review, researchers detail the development history of Principal Editing (PE), the latest evolution of CRISPR-Cas-based technologies. PE was proposed by a team of researchers in 2019, which is characterized by the absence of double-strand breaks (DSBs) or homology sequence patterns with variable application scenarios, including point mutations as well as insertions or deletions. The PE system consists of two parts: the master editors (PEs) and the master editing guide RNA (pegRNA). This PE system has developed and progressed rapidly over the last four years, with versatile advances in its architecture to increase editing efficiency, targeting and specificity, including a new pegRNA design, PE modification and improved delivery. Moreover, despite its relatively recent inception, PE has been widely applied to correct pathological mutations associated with genetic diseases, both in vitro and in vivo , presenting great potential for advancing the field of gene therapy from bench to bedside.