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gene editing

1,407 papers

#gene editing Open access Sep 2026

High-throughput optimized prime editing-mediated endogenous protein tagging for pooled imaging of protein localization - Supplementary datasets related to Figure 6g

Data related to Figure 5g in the manuscript "High-throughput optimized prime editing-mediated endogenous protein tagging for pooled imaging of protein localization".Representative 10-cell albums for all confidently tagged genes (SupplementaryData2-3) and tagging spacers (SupplementaryData4-7). Single cell images were r...

Henry Sanchez, Deepak C. Mani, Tomer Lapidot et al. · 0 citations
#gene editing Open access Sep 2026

High-throughput optimized prime editing-mediated endogenous protein tagging for pooled imaging of protein localization - Supplementary datasets related to Figure 6g

Data related to Figure 5g in the manuscript "High-throughput optimized prime editing-mediated endogenous protein tagging for pooled imaging of protein localization".Representative 10-cell albums for all confidently tagged genes (SupplementaryData2-3) and tagging spacers (SupplementaryData4-7). Single cell images were r...

Henry Sanchez, Deepak C. Mani, Tomer Lapidot et al. · 0 citations
#gene editing Open access Sep 2026

Animal Model Selection and Deep Phenotyping in Mitochondrial Genetic Diseases: Connecting Human Variants, Multisystem Pathology, and Preclinical Evidence

A multidomain framework is proposed that distinguishes target engagement, molecular correction, downstream pharmacodynamic responses, organ function, and whole-animal or patient-relevant benefit without assuming that effective treatments must improve every domain sequentially.

Nobuya Sasaki, Masaki Watanabe · 0 citations
#gene editing Open access Sep 2026

RNA Editing of DEGS1 Links Genetic Risk to Ceramide Dysregulation and Astrocyte Toxicity in ALS

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease with ∼90% of cases being sporadic. Although genome-wide association studies have identified numerous genetic risk loci, these variants account for only a portion of ALS heritability, suggesting that additional genetic and regulatory mechanisms con...

Alan Guo, S. Lessard, Li-Lu Guo et al. · 0 citations
#gene editing Review Sep 2026

Beating the heart failure epidemic: can emerging cell therapies deliver what conventional approaches cannot?

INTRODUCTION Heart failure (HF) is one of the most urgent unmet challenges in cardiovascular medicine. Despite advances in guideline-directed medical therapy (GDMT), substantial morbidity and mortality remain high in patients with advanced disease. This suggests a need for disease-modifying interventions that address u...

Russell G. Saltzman, K. Asensi, Arnab Chakraborty et al. · 0 citations
#gene editing Open access Sep 2026

Combinatorial CRISPR/Cas12a excises targeted loci in the mammalian genome at very low efficiency

Findings indicate that the Cas12a system does not yield locus excision at the efficiency required for sensitive negative selection assays, and showed no meaningful correlation between gRNA efficiency, orientation, GC content, or cut site geometry and the success of locus excision.

N. Anvar, Zsofia M. Szegletes, R. Steger et al. · 0 citations
#gene editing Open access Sep 2026

Genomic Engineering of Gene Dosage: A Generalizable Framework for Modeling Haploinsufficiency-Mediated Human Disorders through Splicing Modulation

Heterozygous loss-of-function variants causing gene dosage reduction underlie many human genetic disorders, yet preclinical mouse models frequently fail to recapitulate human disease phenotypes due to post-translational compensation. Here, we present a generalizable framework to modulate gene dosage by alternative spli...

Eric D. Smith, Joshua K. Meisner, Abbey Bullard et al. · 0 citations
#gene editing Open access Sep 2026

Mutant ASXL1 Cooperates with MECOM and BRD4 to Drive Transcriptional Dysregulation in Human Hematopoiesis.

Mutations in the epigenetic regulator ASXL1 are common in myeloid malignancies and portend a near-universally poor prognosis. While multiple mechanisms for mutant ASXL1-dependent oncogenesis have been proposed, none have been functionally validated in the context of the human hematopoietic stem cell, where these mutati...

Madison L. Hall, Aliya Quintal, Sonia N. Acharya et al. · 0 citations
#gene editing Sep 2026

Abstract PR004: Characterization of patient-specific CHEK2 genetic variants in high-risk neuroblastoma

Roles for certain genetic variants in CHEK2 in NB are supported, as well as potential mechanistic targets for high-risk or therapy-resistant NB, and preclinical information is provided to inform diagnostics and potential therapeutic strategies for high-risk NB.

Xue-Ting Xiong, Meagan Beffort, L. Kee et al. · 0 citations
#gene editing Dataset Open access Sep 2026

Supporting data for “Transfer RNA Abundance and Modification Shape Codon-Dependent Translational Adaptation in Hepatocellular Carcinoma”

This dataset was generated as part of a research project investigating how transfer RNA (tRNA) abundance and tRNA modification regulate codon-dependent translation in hepatocellular carcinoma (HCC). The data cover two complementary aspects of tRNA-mediated translational regulation.The first project examines the relatio...

Yujie Gan · 0 citations
#gene editing Open access Sep 2026

Chitinase variants establish environmental inflammatory lung conditioning

Environmental particulate exposure contributes to the severity and persistence of numerous acute and chronic lung diseases, including asthma, viral pneumonia, acute respiratory distress syndrome, and pulmonary fibrosis. Chitin, a highly abundant environmental polysaccharide present in fungi, insects, and mites, is cont...

Yilin Wang · 0 citations

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Google DeepMind Blog Sep 30, 2026

Introducing SynthID Bio

Proof of concept for watermarking AI-generated proteins while preserving biological function.

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