gene editing

Atypical MDM2 p53 Regulation and Chemosensitivity Induced by Proximal PAS Deletion

Results suggest that MDM2 3’UTR elongation may trigger a non-canonical regulatory mechanism that bypasses the traditional MDM2-p53 interaction, and suggests that APA-mediated gene modulation can induce unforeseen compensatory survival pathways in cancer cells, necessitating further investigation into the broader functional landscape of elongated 3'UTRs.

Generation of two homozygous iPSC lines carrying variants of uncertain significance in LMNA associated with cardiomyopathy.

Two isogenic induced pluripotent stem cell lines carrying homozygous LMNA variants are generated by prime editing of a healthy donor iPSC line, providing a valuable platform for functional characterization and potential clinical reclassification of LMNA VUS.

Design of base editing systems with tunable editing hotspots within a defined window through sgRNA engineering.

Base editors (BEs) enable efficient A-to-G or C-to-T conversions without double-stranded DNA cleavage, but their editing windows remain difficult to tune, limiting genome engineering flexibility. Here, we engineered CRISPR/Cas12b sgRNA by introducing MS2 hairpins to recruit an MS2-N55K-cytidine deaminase-UGI complex, enabling programmable control of the editing window. Three modified sgRNAs were generated by replacing two loop regions, each producing distinct editing hotspots in E. coli. The AID*Δ-MSBE system (sgRNA1.1) generated a window near the PAM with peak activity at C7-C9, while the CDA-MSBE system (sgRNA1.2) produced a distal window with peak activity at C20-C23. Both systems exhibited identical editing patterns in Bacillus subtilis. A dual-orthogonal system (MS2 and PP7) was constructed to simultaneously recruit two deaminase complexes, restoring the classic dCas12b CBE editing pattern. Rifampicin resistance assays confirmed high targeting specificity with low off-target effects. As proof of concept, the MSBEs were successfully employed for the flexible reprogramming of sfGFP fluorescence and the targeted evolution of the endogenous gene rpsE, respectively. Collectively, we developed the MSBEs with tunable editing hotspots, providing innovative tools to enhance the flexibility and accessibility of BEs for genome engineering.

Engineering HSV-1 for Gene Therapy, Oncolytic Immunotherapy, and Neuroscience.

This review illustrates how HSV-1 has evolved from a naturally neurotropic virus into a versatile biomedical tool, whose therapeutic and research potential emerges from the precise matching of viral properties with disease-specific requirements, delivery contexts, and functional objectives.

Can host genetics transform the sustainable control of tropical theileriosis? Insights from the Tick-Theileria interface.

The evidence indicates that host genetics will not replace existing control but could reduce reliance on acaricides and chemotherapy, and the evidence indicates that host genetics will not replace existing control but could reduce reliance on acaricides and chemotherapy.

Gene editing of alveolar organoids reveals AT2 dysfunction due to endosomal SFTPC accumulation.

Endosomal dysfunction and polarity loss are established as drivers of SFTPC-I73T-mediated epithelial injury and mechanisms that may underlie AT2 dysfunction in disease more broadly are highlighted.

Global genetic modifiers of fetal hemoglobin in sickle cell disease: Systematic review and meta-analysis.

Evidence on genetic determinants of Hb F and quantified effects of key variants through meta-analysis is synthesized, supporting genotype-guided therapeutic, and genome-editing strategies.

CRISPR gene therapy: a review

Clinical applicability is limited by issues such off-target effects, PAM sequence restrictions, DNA damage-induced toxicity, and immunological responses to Cas proteins, despite its wide therapeutic potential, but improvements in delivery methods and high-fidelity Cas9 variations are being addressed.

Intranasal CRISPR lipid nanoparticles targeting MAPK9 attenuate neuroinflammation after traumatic brain injury

It is demonstrated that intranasal delivery of Iba-1-targeted CRISPR-LNPs enables effective MAPK9 modulation in Iba-1 + myeloid cells within the injured brain and attenuates acute neuroinflammation following TBI.

Eimeria in chickens: Recent advances in host-pathogen interactions, molecular insights, and control strategies.

This review systematically synthesizes key advances over the past decade concerning host-Eimeria interactions, molecular regulatory mechanisms, and novel control strategies, while contextualizing these findings with earlier seminal discoveries.

Gene Editing Therapy Developer Scribe Therapeutics Closes $155.5M IPO.

Genome-wide Identification of CXE Gene Family in Soybean and Functional Characterization of GmCXE31 in Lipid Biosynthesis and Salt Tolerance.

This study elucidates the multifaceted roles of GmCXE31 in coordinating soybean salt tolerance, lipid metabolism and agronomic traits, providing theoretical and genetic resources for salt-tolerant and high-quality soybean molecular breeding.