gene editing

The utilitarian temptation in gene editing.

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...

Gene-Targeted Therapies in Inherited Bone Marrow Failure Syndromes

Inherited bone marrow failure syndromes (IBMFS) are genetically heterogeneous multiorgan disorders sharing defective hematopoiesis and cancer predisposition. Here, we examine emerging strategies to suppress nonsense mutations, with a focus on translational readthrough-inducing drugs (TRIDs), nonsense-mediated decay mod...

Functional Improvement of dystrophic muscle by utrophin gene editing

Streamlining oversight in gene-editing trials.

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...

Reactivation of the embryonic ζ-globin gene ameliorates severe forms of α-thalassemia

Hemoglobinopathies are the most common inherited anemias worldwide. While most carriers are asymptomatic or mildly affected, severe forms of α-thalassemia, including severe hemoglobin H (HbH) disease and surviving individuals with Hb Bart’s hydrops fetalis syndrome (BHFS), require lifelong transfusion support or stem c...

LNP-mediated CRISPR gene editing of human vascular endothelial cells in 2D and 3D microfluidic cell culture systems

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...

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...

Precision Genome Engineering: Clinical Translation, Delivery Architectures, and Commercial Evolution in Gene Editing

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...