gene editing

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...

Clean gene technology for precision breeding and genetic improvement of vegetable crops

This paper summarizes the fundamentals, use, and future opportunities of clean gene technology for improving essential characteristics of vegetable crops, such as yield, quality, resistance to diseases, and tolerance to abiotic stress, as well as the regulatory and social environment.

Digital Genome Workstation: interactive and non-destructive editing of genome variants

Genome-variant analysis is commonly organized as linear pipelines for annotation, filtering, and prioritization. Digital Genome Workstation (DGW) complements these workflows with a local desktop environment for interactively constructing, editing, and comparing alternative diploid variant configurations derived from a...

A highly efficient detached-leaf transient expression system and endogenous promoter toolkit for advancing gene functional analysis and gene editing in Cymbidium

transient expression system and endogenous promoter toolkit for advancing gene functional analysis and gene editing in Cymbidium, aBIOTECH,

A Qualitative and Quantitative Detection Method for Gene Editor Residues Based on Cutting-Edge Biosensing Technology

This study focuses on the core pain points of the current gene editing industry, specifically addressing the residual editor issue arising from the widespread application of CRISPR/Cas9 technology. A novel, highly sensitive method for the qualitative and quantitative detection of residual Cas9 has been developed, which...

Editing cells in place: Targeted in vivo gene editing of hematopoietic stem cells.

Traditional ex vivo hematopoietic stem cell (HSC) gene therapy faces manufacturing challenges. A recent study1 in Nature Biomedical Engineering demonstrates that antibody-engineered lipid nanoparticles achieve efficient and durable in vivo editing of HSCs in humanized mice, offering a promising therapeutic strategy for...

The utilitarian temptation in gene editing.