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gene editing

1,561 papers

#gene editing Oct 2026

Base Editing as a Therapeutic Strategy for Autosomal Dominant Tubulointerstitial Kidney Disease caused by Pathogenic UMOD Variants

Autosomal dominant tubulointerstitial kidney disease caused by pathogenic UMOD variants (ADTKD-UMOD) is an untreatable monogenic kidney disease in which misfolded uromodulin is retained in the endoplasmic reticulum and exerts a toxic gain-of-function effect. Here we systematically evaluate the full spectrum of base edi...

Nora Diederich, Sarah Wendlinger, Shuhan Kong et al. · 0 citations
#gene editing Open access Oct 2026

The Genetic Discovery (GeDi) resource: Deep characterization of a multi-ancestry panel of human reference iPSCs for functional genomic studies

Human induced pluripotent stem cells (iPSCs) are essential tools for disease modelling, drug discovery, and functional genomics, yet the field remains anchored to a small number of reference lines, limiting our understanding of how genetic background shapes cellular phenotypes across human populations. Here we describe...

Adrian Melo-Carrillo, Enrica Pellegrino, Parithi Balachandran et al. · 0 citations
#gene editing Open access Oct 2026

VASP cooperates with zyxin to organize mechanically competent adhesions

Abstract Focal adhesions (FAs) are dynamic macromolecular assemblies that anchor the actin cytoskeleton to the extracellular matrix via integrin receptors, thereby regulating cell morphology and migration. Here, we show that the vasodilator-stimulated phosphoprotein (VASP) cooperates with the mechanosensitive scaffold...

Wen-Lu Chung, Rajaa Boujemaa‐Paterski, Sabina E. Winograd-Katz et al. · 0 citations
#gene editing Open access Oct 2026

Development of an Efficient In Vitro Propagation Protocol for the Elite Bidah Pomegranate (Punica granatum L.) Landrace in Saudi Arabia

The Bidah pomegranate (Punica granatum L.) is an elite landrace native to Wadi Bidah in the Al-Baha region of Saudi Arabia. This cultivar is distinguished by its unique taste, high yield, genetic component, and phytochemical profile. In addition, its resilience to harsh environmental conditions aligns closely with nati...

Nada M. Alattas, Khalid M. Al-Rohily, Saheed O. Anifowose et al. · 0 citations
#gene editing Open access Oct 2026

CRISPR/Cas9 induced disruption of oca2 and tyr genes associated with pigmentation in Danio rerio

Targeted disruption of genes has been successfully carried out in several model and non-model fish species. In ornamental fish industries, Danio rerio (D. rerio) represents the most significant fish species and is available in several color varieties. Recently, CRISPR/Cas9 has been used to disrupt pigmented genes in se...

Kriti Kumari, Gowhar Iqbal, S. S. Priyanka et al. · 0 citations
#gene editing Open access Oct 2026

Functional dissection of ultraconserved non-coding regions present near the Hoxd complex in vertebrates

Ultraconserved non-coding elements (UCEs) exhibit near-perfect sequence conservation across vertebrate genomes, implying severe evolutionary constraint and essential biological functions. Near the vertebrate Hoxd complex, a master regulator of axial patterning and limb morphogenesis, three Conserved Regions (CR1, CR2,...

Sonu Yadav, Anil Kumar, Rakesh K. Mishra · 0 citations
#gene editing Open access Oct 2026

Artificial Intelligence as a Catalyst for Cell-Based Therapies in Retinal Disease

Retinal degenerative diseases, including age-related macular degeneration (AMD), retinitis pigmentosa (RP), and Stargardt disease, represent leading causes of irreversible vision loss worldwide, collectively affecting hundreds of millions of people. Cell-based therapies, particularly those leveraging induced pluripoten...

Girish Kumar Srivastava, Priyank Shukla, Sara Crespo Millas et al. · 0 citations
#gene editing Open access Oct 2026

Rapid development of durable bacterial blight resistance in African rice through marker-assisted pyramiding of four resistance genes

Abstract Bacterial blight of rice causes substantial crop losses in Asia and Africa. The recent introduction of Asian strains into African countries has led to two independent outbreaks detected in 2019, causing severe damage in Madagascar and Tanzania. The strains are highly virulent on local rice varieties and rapidl...

Yugander Arra, Eliza P. I. Loo, Camille Blasco et al. · 0 citations
#gene editing Open access Oct 2026

Evolutionary Diversification and Functional Roles of MYB Transcription Factors in Algae: Insights from Genomic Distribution and Regulatory Networks

MYB transcription factors (TFs) represent a conserved family of DNA-binding regulators involved in diverse cellular and physiological processes. MYB TFs are well-characterized in terrestrial plants, but their evolutionary history and functional diversity in algae remain poorly understood. This review summarizes current...

Yurong Wang, Chunli Luo, Xunmeng Li et al. · 0 citations
#gene editing Book Open access Oct 2026

Programmable Enzymes for Large-Cargo Genome Engineering: From Targeted DNA Integration to Therapeutic Genome Writing

Genome engineering is undergoing a conceptual transition from the correction of individual sequence variants toward the programmable installation of larger functional genetic elements. CRISPR-associated nucleases established RNA-guided genome targeting as a broadly accessible technology, while base and prime editing su...

Hatice Esenkaya · 0 citations
#gene editing Open access Oct 2026

Retrotransposons: A New Frontier in In Vivo Genome Engineering

Genome engineering has transformed cell therapy, but clinical implementation remains largely confined to ex vivo workflows followed by adoptive cell transfer. These approaches require costly manufacturing, often rely on viral vectors, and carry risks associated with unwanted genomic alterations. Recent advances in lipi...

Philippe Kieffer-Kwon, Meghan T. Harris, Daniel R. Getts et al. · 0 citations
#gene editing Oct 2026

High-Throughput Screening of a Lipid Nanoparticle (LNP) Library Identifies Lead Formulations for CRISPR-Cas9 Ribonucleoprotein Delivery and Gene Editing.

Abstract Delivery of CRISPR-Cas9 ribonucleoprotein (RNP) using synthetic materials can bypass the host translational machinery and limit the duration of genomic exposure, minimizing the risk of off-target mutations. Despite their advantages, lipid nanoparticle (LNP) platforms for CRISPR-Cas9 RNP delivery that achieve h...

Yu Wang, Amin Haghighat Naeini, Nishant Kumar et al. · 0 citations

From tech blogs

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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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