gene editing

On the physics of chromatin molecular complex: from phase-separation to membrane adsorption

Genome organization within the cell nucleus is closely linked to essential cellular functions, including gene expression, DNA replication and DNA repair (Dekker and Mirny, 2024;Willemin et al., 2024;Bantele et al., 2025;Dekker et al., 2025). It is therefore crucial to understand the rules that dictate how chromatin thr...

mRNA Lipid Nanoparticle Delivery Systems: Composition, Optimization and Advances in Clinical Translation

Lipid nanoparticles (LNP) represent critical non‑viral carriers for mRNA delivery, capable of encapsulating, protecting mRNA, facilitating cellular uptake and enabling cytosolic release. This review summarizes the composition and delivery mechanisms of mRNA‑LNP. Optimization strategies including ionizable lipid structu...

Somatic Gene‑Editing Potential and Engineering Barriers for Chronic Cardiovascular Disease Management

Cardiovascular diseases (CVDs) are major chronic health conditions, and some patients require long-term medication to control disease progression and associated risks. The need for more durable treatment has therefore significantly increased, leading to importance to find a more long-lasting treatment strategy. This pa...

Estrogen and Alzheimer’s Disease: Neurobiology, Epigenetics, and AI-Driven Therapeutic Design

Alzheimer’s disease (AD) is a progressive neurodegenerative disorder, and women are disproportionately affected. This review examines the evidence that estrogen signaling contributes to this disparity and asks what a molecularly targeted therapeutic would require. We first distinguish prevalence from incidence in the g...

Epigenetics as a design variable in heterologous expression

ABSTRACT Heterologous expression—the expression of genes from one organism into another—is an important approach to produce value-added chemicals such as proteins and metabolites. The main challenge, however, is achieving high production yields that are commercially viable. Approaches used to improve productivity rely...

Impacts of different SETD1A mutations and genetic backgrounds on human induced pluripotent stem cell-derived hippocampal neurons

Human induced pluripotent stem cells (hiPSCs) provide a translational platform to interrogate the impact of genetic risk factors for various disorders on human biology. It has been a common practice to engineer premature stop codons to model predicted loss-of-function mutations using isogenic hiPSCs, yet effects of mut...

CRISPR/Cas9-based nanodelivery systems for precision gene editing in neuroinflammation

The potential of integrating CRISPR/Cas9 with nanodelivery systems to advance precision medicine for neuroinflammatory disorders, while emphasizing their potential for therapeutic applications in complex CNS conditions is highlighted.

Author Correction: UNCOVERseq enables sensitive and controlled gene editing off-target nomination across CRISPR-Cas modalities and systems

In the version of the article initially published, the graphs in Fig. 2a were missing and have now been reinserted in the HTML and PDF versions of the article.

Next-generation pharmacological strategies for Parkinson’s disease: a systematic literature review of symptom management and disease modification

Septins regulate cytokinesis and multicellular development in the closest living relatives of animals

ABSTRACT Septins are cytoskeletal proteins that regulate cytokinesis in fungi and animals, yet their functions in choanoflagellates — the closest living relatives of animals — have remained unknown. Salpingoeca rosetta, a choanoflagellate that switches between unicellular and multicellular forms, encodes four septins c...

A cryptic binding pocket regulates the metal-dependent activity of Cas9

Abstract Cas9 is a metal-dependent nuclease used for genome editing across diverse cells and organisms exhibiting distinct ionic environments, yet how metal ions regulate its catalytic function, and consequently its editing efficiency remains unclear. Here, molecular simulations, Markov state models, and mixed quantum–...

Advances in genetically engineered and humanized mouse models for dissecting sepsis mechanisms and therapeutic response.

Progress in mouse genetics, CRISPR/Cas-based genome editing, conditional alleles, and next-generation humanized platforms has expanded the toolkit for functional interrogation of host pathways that shape sepsis outcomes, and can link genotype-to-phenotype mechanisms with biomarker-informed therapeutic testing.