gene editing

Repurposing Cas12a2-mediated abortive infection for efficient and low-toxicity microbial genome editing

CRISPR-Cas systems have revolutionized microbial genome engineering, yet counter-selection strategies typically rely on targeted DNA cleavage, where the severe cytotoxicity of double-strand breaks restricts transformation efficiency and clonal recovery. Here, we present an innovative strategy, SuCas12a2-Universal Recom...

Dynamic phosphorylation of DGCR8 regulates embryonic stem cell differentiation and microprocessor function

The Microprocessor complex, minimally composed of DROSHA and DGCR8, is ubiquitously required for microRNA biogenesis. It also regulates the balance between pluripotency and differentiation through mRNA alternative splicing and mRNA cleavage. However, how these various activities are regulated remains incompletely und...

PTPN22 as a novel therapeutic target: a key intracellular checkpoint for NK cell therapy

Abstract Background PTPN22, a protein tyrosine phosphatase (PTP) family member, has recently emerged as an intracellular regulator in adaptive immune cells. However, its function in natural killer (NK) cells remains unclear despite its high expression in innate immune cells. Methods We analyzed single-cell RNA-sequenci...

Identification of Novel Regulators of Telomere Length and its Impact on Endothelial Dysfunction

Telomere length (TL) is highly heritable and associated with a plethora of health conditions, including cancer, Alzheimer's and coronary artery disease (CAD). A number of studies have linked TL to CAD, with short TL preceding disease onset. Genetic studies support a causal link between the two, though the mechanism rem...

Post-transcriptional regulation of Influenza A virus mRNAs

The fate of cellular mRNAs is governed by intricate and dynamic post-transcriptional regulatory mechanisms that fine-tune cellular gene expression. Processes such as stabilisation, degradation, splicing, nuclear export and RNA editing, are essential for mRNA processing and are known to be hijacked by RNA viruses to enh...

Immuno-metabolic crosstalk in osteoarthritis: from mechanisms to therapies

Osteoarthritis (OA) is a heterogeneous whole-joint disorder influenced by mechanical stress, aging, previous trauma, genetic susceptibility, subchonrral bone alterations, and localized inflamation.Emerging evidence suggests that immune and metabolic processes alterations may be involved in OA pathogenesis particula...

Should ApoE4 gene editing therapy be approached with caution when AD is accompanied by retinal neurodegenerative diseases?

Apolipoprotein E4 (ApoE4) is the strongest genetic risk factor for Alzheimer’s disease (AD), and ApoE4-directed gene editing and gene therapy have emerged as promising therapeutic strategies for AD, although they remain investigational and are not yet an established mainstream treatment. Interestingly, however, ApoE4 i...

Tissue-aware genetic prioritization across autoimmune and proliferative thyroid diseases: analysis code and derived data

Journal-independent reproducibility archive for tissue-aware genetic prioritization across autoimmune and proliferative thyroid diseases. Version 1.1.1 aligns Tier 2 with the implemented external replication or consistency criterion, separately records exact phenotype-matched MVP evidence, and corrects the MVP flags fo...

Acid-Activatable Rolling Circle Replication-Based Nucleic Acid Nano-Prodrug for Combined Gene Therapy of Pancreatic Cancer.

Nucleic acid-based gene therapeutics hold great potential for precisely treating pancreatic ductal adenocarcinoma (PDAC), yet their clinical translation remains challenged by the lack of efficient delivery platforms capable of simultaneously minimizing payload leakage, intrinsic toxicity, and immunogenicity. Herein, we...

Chimeric antigen receptor (CAR) therapies in retinoblastoma: A comprehensive review

Retinoblastoma (RB) is the most common malignant intraocular tumor in children and remains a major concern in pediatric ophthalmology, particularly in advanced, metastatic, or treatment-resistant cases. Although current treatment approaches have greatly improved survival in high-income countries, important limitations...

Epistemic justice in gene-editing research.

Environmental adaptation in wheat landraces: integrating physiological, molecular, and ecological mechanisms for climate-resilient agriculture

Climate change poses an increasing threat to wheat production, particularly in arid and semi-arid regions where rising temperatures, water scarcity, soil degradation, and salinity interact to reduce crop productivity and stability. Wheat landraces constitute an invaluable reservoir of adaptive genetic diversity that ha...