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gene editing

1,680 papers

#gene editing Review Open access Sep 2026

Decoding MAPT Exon 10 Mis-Splicing in FTDP-17: From Pathogenic Mechanisms and Experimental Models to Molecular Therapies

This review explores the molecular mechanisms regulating exon 10 splicing, focusing on how exonic and intronic splicing mutations destabilise mRNA structures or alter splicing factor binding, and discusses how these changes can cause disease symptoms in experimental models.

Giuseppina Covello · 0 citations
#gene editing Sep 2026

Transcriptome Profiling of RNA Editing in Mitochondrial nad Genes of Catharanthusroseus: Organ-Specific Patterns and Functional Implications

Abstract RNA editing is a major post-transcriptional process in plant organelles that frequently alters coding sequences and can influence mitochondrial gene function. However, organ-resolved RNA editing in mitochondrial nad transcripts remains poorly characterized in Catharanthus roseus (L.) G.Don, despite the biologi...

A. A. Alnufaei · 0 citations
#gene editing Open access Sep 2026

Comparative analysis of organelle genomes in three Terminalia species reveals structure evolution and phylogenetic position

Plant organelle genomes are important for evolutionary biology research, yet remain poorly characterized in genus Terminalia (Combretaceae). Here, we systematically analyzed the mitochondrial genomes (mtDNA) and chloroplast genomes (cpDNA) of three Terminalia species ( Terminalia chebula , Terminalia franchetii , Termi...

Xingyu Liu, Yingmin Zhang, Pengrui Luo et al. · 0 citations
#gene editing Sep 2026

BDF6 Deficiency Severely Compromises Intracellular Amastigote Development and Infectivity of Trypanosoma cruzi

Abstract Trypanosoma cruzi, the causative agent of Chagas disease, relies on complex gene regulatory mechanisms to adapt to its diverse host environments. In recent years, it has been established that epigenetics plays an essential role in these mechanisms via the regulation of chromatin structure. Bromodomain-containi...

Victoria Boselli Raposo, Virginia Perdomo, Florencia Pacini et al. · 0 citations
#gene editing Review Sep 2026

Targeted Epigenetic Reactivation strategies as new treatments for Prader-Willi Syndrome: Achievements and Challenges.

Key translational challenges are discussed, focusing on the imperative to optimize delivery vectors for widespread central nervous system distribution, the necessity of advanced human-derived neuronal models to validate functional recovery, and the critical need to prevent iatrogenic Angelman Syndrome caused by UBE3A i...

S. Alberti, Irina Cutei, Giacomo Costa Broccardi et al. · 0 citations
#gene editing Open access Sep 2026

p38 MAPK inhibition safeguards genome integrity during gene editing of hematopoietic stem and progenitor cells.

Ex vivo genome editing of hematopoietic stem and progenitor cells (HSPCs) holds significant therapeutic potential but remains constrained by genotoxic risks associated with nuclease-induced DNA double-strand breaks, DNA donor template delivery and sensing, and proliferation-induced stress during ex vivo manipulation. T...

Roberta Vacca, Lucrezia della Volpe, Nicolò Gualandi et al. · 0 citations
#gene editing Open access Sep 2026

Advance in Life Sciences: Present and Future – A Research Perspective

ABSTRACT Life sciences represent one of the most dynamic and transformative scientific domains in the modern world. Rapid technological progress, interdisciplinary research integration, and molecular-level understanding of biological systems have revolutionized healthcare, agriculture, environmental protection, and bio...

Adi Reddy Rakkireddy · 0 citations
#gene editing Dataset Open access Sep 2026

Supporting data for "Uncovering cellular response to ionizable lipid-induced endolysosomal damage with multimodal imaging"

Lipid nanoparticles (LNPs) are the leading non-viral platform for nucleic acid delivery, validated clinically by their use in mRNA vaccines and the siRNA drug patisiran. Nevertheless, their wider use in protein-replacement and gene-editing therapeutics need repeated doses is limited by inflammatory side effects caused...

Gu Chen · 0 citations
#gene editing Review Open access Sep 2026

Zebrafish in the era of single-cell and spatial transcriptomics: a new paradigm for central nervous system drug discovery from natural products.

Combined with CRISPR-based gene editing and phenotypic assays, this integrative approach positions zebrafish as a cornerstone of modern pharmacology and a transformative platform for CNS therapeutics.

Yida Huang, Xiang Liu, Guang-Yi Fan 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

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

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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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