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The discovery and application of genome editing introduced a new era of plant breeding by giving researchers efficient tools for the precise engineering of crop genomes. Here we demonstrate the power of genome editing for engineering broad-spectrum disease resistance in rice (Oryza sativa). We first isolated a lesion mimic mutant (LMM) from a mutagenized rice population. We then demonstrated that a 29-base-pair deletion in a gene we named RESISTANCE TO BLAST1 (RBL1) caused broad-spectrum disease resistance and showed that this mutation caused an approximately 20-fold reduction in yield. RBL1 encodes a cytidine diphosphate diacylglycerol synthase that is required for phospholipid biosynthesis. Mutation of RBL1 results in reduced levels of phosphatidylinositol and its derivative phosphatidylinositol 4,5-bisphosphate (PtdIns(4,5)P). In rice, PtdIns(4,5)P is enriched in cellular structures that are specifically associated with effector secretion and fungal infection, suggesting that it has a role as a disease-susceptibility factor. By using targeted genome editing, we obtained an allele of RBL1, named RBL1, which confers broad-spectrum disease resistance but does not decrease yield in a model rice variety, as assessed in small-scale field trials. Our study has demonstrated the benefits of editing an LMM gene, a strategy relevant to diverse LMM genes and crops.
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http://dx.doi.org/10.1038/s41586-023-06205-2 | DOI Listing |
Mol Ther
September 2025
Be Biopharma, Cambridge, MA, 02139, USA. Electronic address:
Hemophilia B gene therapy treatments currently have not addressed the need for predictable, durable, active, and redosable factor IX (FIX). Unlike conventional gene therapy, engineered B Cell Medicines (BCMs) are durable, redosable, and titratable, and thus have the potential to address significant unmet needs in the Hemophilia B treatment paradigm. BE-101 is an autologous BCM comprised of expanded and differentiated B lymphocyte lineage cells genetically engineered ex vivo to secrete FIX-Padua.
View Article and Find Full Text PDFClin Transl Oncol
September 2025
Department of Basic Science, College of Medicine, Princess Nourah bint Abdulrahman, University, P.O.Box 84428, 11671, Riyadh, Saudi Arabia.
Esophageal cancer (EC) is one of the most serious health issues around the world, ranking seventh among the most lethal types of cancer and eleventh among the most common types of cancer worldwide. Traditional therapies-such as surgery, chemotherapy, and radiation therapy-often yield limited success, especially in the advanced stages of EC, prompting the pursuit of novel and more effective treatment strategies. Immunotherapy has emerged as a promising option; nonetheless, its clinical success is hindered by variable patient responses.
View Article and Find Full Text PDFInt J Biol Macromol
September 2025
Plant Biotechnology Research Center, Fudan-SJTU-Nottingham Plant Biotechnology R&D Center, School of Agriculture and Biology, Shanghai Jiao Tong University, Minhang, Shanghai, 200240, China. Electronic address:
Vitamin A deficiency is one of the most severe micronutrient-related health issues worldwide. Tomatoes, a widely cultivated crop for their adaptability, nutritional value, and lycopene content (a beta-carotene precursor), are ideal candidates for biofortification. In this study, CRISPR-mediated knockout mutants (cr-SlLCYe and cr-SlBCH) were generated to enhance the precursor supply to the β-carotene biosynthetic pathway and reduce its degradation.
View Article and Find Full Text PDFNeurogenetics
September 2025
Nur International University, 54600, Lahore, Punjab, Pakistan.
Huntington's disease (HD) is a progressive, autosomal dominant neurodegenerative disorder characterized by motor dysfunction, cognitive decline, and psychiatric disturbances. It is caused by CAG repeat expansions in the HTT gene, resulting in the formation of mutant huntingtin protein that aggregates and disrupts neuronal function. This review outlines the pathogenesis of HD, including genetic, molecular, and environmental factors.
View Article and Find Full Text PDFNat Genet
September 2025
Bioinformatics Interdepartmental Program, University of California, Los Angeles, CA, USA.
Gene expression is modulated jointly by transcriptional regulation and messenger RNA stability, yet the latter is often overlooked in studies on genetic variants. Here, leveraging metabolic labeling data (Bru/BruChase-seq) and a new computational pipeline, RNAtracker, we categorize genes as allele-specific RNA stability (asRS) or allele-specific RNA transcription events. We identify more than 5,000 asRS variants among 665 genes across a panel of 11 human cell lines.
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