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gene editing

1,554 papers

#gene editing Open access Oct 2026

A systematic fluorescently tagged protein toolkit for dissecting apical extracellular matrix patterning in C. elegans.

Barrier epithelia are shielded from the external environment by their apical extracellular matrices (aECMs). The molecular complexity of aECMs has challenged understanding of their organization in vivo. To define the molecular architecture of a model aECM we generated a toolkit of 105 fluorescently tagged aECM componen...

J. M. Ragle, Murugesan Pooranachithra, Guinevere A. Ashley et al. · 0 citations
#gene editing Open access Oct 2026

Poison exon splicing in the human brain provides a new paradigm for understanding and targeting neurological disorders

Poison exons are highly conserved exons whose inclusion creates premature termination codons and triggers nonsense-mediated decay (NMD) of the mature transcript. Despite their important role in post-transcriptional regulation, poison exons remain poorly annotated due to the lack of systematic transcriptome-wide approac...

Paolo Pigini, Huilin Xu, Yan Ji et al. · 0 citations
#gene editing Review Open access Oct 2026

Retinitis Pigmentosa: A Review of Its Pathogenesis and Emerging Molecular and Cellular Therapies

Retinitis pigmentosa (RP) is the most common inherited retinal dystrophy and remains largely untreatable. This review summarizes RP pathogenesis and critically appraises emerging therapeutic strategies, including gene augmentation, gene editing, RNA-based therapies, gene-agnostic and neuroprotective approaches, stem-ce...

Nicolas Tuli, Moath Baeshen, Mohamed Echbicheb et al. · 0 citations
#gene editing Review Oct 2026

Evolutionary Genetics of Insect Mimicry and Masquerade.

As classic examples of adaptive evolution, insect mimicry and masquerade have been studied extensively for more than a century. Both phenomena protect insects from predators and are widely observed in nature. Previous research on phenotypic, genetic, phylogenetic, and behavioral aspects has laid a solid foundation for...

Yu-Bo Zhang, Wei Zhang · 0 citations
#gene editing Open access Oct 2026

Edited HSPCs rescue frataxin deficiency and inflammation-driven multisystemic pathology in Friedreich's ataxia mice.

Friedreich's ataxia (FRDA) is a multisystemic neurodegenerative disorder caused by GAA repeat expansion in intron 1 of frataxin (FXN), resulting in FXN deficiency and mitochondrial dysfunction. Current therapies do not correct the underlying genetic defect. Here, we demonstrate that ex vivo CRISPR/Cas9 excision of the...

Anusha Sivakumar, Alexis N. Corl, Keneth Jordan Muñoz et al. · 1 citation
#gene editing Oct 2026

A Standalone Flavoprotein Monooxygenase Directs Divergent Tropolone Skeletal Editing through Baeyer–Villiger-Triggered Rearrangement

Abstract Late-stage oxidative remodeling of tropolone cores underpins the remarkable scaffold diversity in fungal meroterpenoid biosynthesis. However, the mechanistic basis by which a single enzymatic catalyst diverts a common substrate toward carbon-excising ring contraction versus carbon-retentive skeletal rearrangem...

Ziang Chen, Jiawang Liu, Songlin Bai et al. · 0 citations
#protein folding Open access Oct 2026

LNP Delivery of Prime Editors Enables Durable Correction of PAH R408W in Phenylketonuria Models

Phenylketonuria (PKU) is a monogenic metabolic disorder caused by mutations in the phenylalanine hydroxylase (PAH) gene, the most common of which is c.1222C>T (p.R408W). Current treatment options, including dietary restriction and enzyme substitution, can be effective but are often challenging for patients to maintain...

S. Yazinski, S. Canali, Yang Xu et al. · 0 citations
#gene editing Open access Oct 2026

Generation of Elite Ideal Rice Germplasm via CRISPR/Cas9-Mediated Multiplex Targeted Gene Editing.

Gene editing of five key negative regulatory genes, GS3 , OsBADH2 , Xa13 , Bsr‐d1 and OsSULTR3;6 , generated an elite ideal‐type rice variety mk‐10 , which exhibits a longer grain shape, enhanced tolerance to heat and alkali stress, increased 2AP content, and simultaneous resistance to Xoo , rice blas...

Jiang-Min Xu, Jian-Min Li, Yi-Lin Luo et al. · 0 citations
#gene editing Open access Oct 2026

The Role of Immunotherapy and Gene Therapy in The Treatment of Breast Cancer in Middle-Aged Women: Integration with Pathomorphological Markers and Organ-Preserving Surgery

This article analyses modern, biologically targeted approaches to the treatment of breast cancer in middle-aged women. The main focus is on determining the pathomorphological and molecular characteristics of the tumour, the role of immunotherapy, particularly in triple-negative breast cancer, the current level of clini...

Dadamukhamedova Khalida Erkinovna, Begimqulov Mirjalol Yashin ugli, Kuvondikova Zulfizar Jasurbek qizi · 0 citations
#gene editing Open access Oct 2026

Precision Gene-Based Therapy for PFIC: Matching Platform to Disease Biology

Progressive familial intrahepatic cholestasis (PFIC) encompasses genetically heterogeneous disorders affecting bile transport, membrane organisation, epithelial polarity and bile-acid signalling. Current medical and surgical treatments can improve symptoms and disease course in selected patients, but substantial unmet...

Ziyu Jiang, S. Tamir Rashid, Paul Gissen et al. · 0 citations
#gene editing Open access Oct 2026

Loss of nono impairs cardiac morphogenesis and excitation–contraction coupling via the PI3K/Akt pathway in zebrafish

Pathogenic variants in NONO are clinically linked to congenital heart disease (CHD), but in vivo mechanisms and gene-targeted interventions remain poorly explored. Using CRISPR/Cas9 gene editing and morpholino silencing, we established knockout (KO) and knockdown (KD) zebrafish models to study nono in cardiogenesis. Ad...

Qiuping Zhang, Chongyang Yan, 刘瀚旻 et al. · 0 citations
#gene editing Open access Oct 2026

Has it been a crime or should it be criminalised? A case study on genome-edited babies and the improvement of criminal statutes in China

The 2018 “gene-edited babies” incident involving He Jiankui elicited widespread global condemnation, and he was subsequently convicted of and sentenced for illegal medical practice. Although several years have passed, impulses and initiatives aimed at advancing the clinical application of human germline genome editing...

Dan Yang · 0 citations

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Microsoft Research Blog Oct 15, 2026

Proof-based general-purpose computing with Magnetar

Zero-knowledge proofs can verify computing results without rerunning workloads or exposing sensitive data. Magnetar helps bring them into the mainstream with support for standard programming languages, major speed gains, and quantum-resistant security. The post Proof-based general-purpose computing with Magnetar appeared first on Microsoft Research.

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