gene editing

Advancing Gene Editing for Children with Hemoglobinopathies.

Highly efficient base editing at PCSK9 and normal human embryo development.

DNA repair outcomes at nicks and mismatches introduced by base editors at the PCSK9 and HBG loci in human embryos are evaluated, finding that unlike Cas9-induced DNA breaks, base editor-induced lesions are efficiently repaired.

A Single-Administration AAV Gene Therapy Expressing an Affinity-Detuned Feline AIM (m-fAIM) for Chronic Kidney Disease in the Domestic Cat: A Theoretical Development Program

This work presents a theoretical development program for a single-administration adeno-associated virus (AAV) gene therapy designed to treat chronic kidney disease (CKD) in the domestic cat. The approach centers on the delivery of an affinity-detuned variant of feline Apoptosis Inhibitor of Macrophage (m-fAIM), enginee...

A Single-Administration AAV Gene Therapy Expressing an Affinity-Detuned Feline AIM (m-fAIM) for Chronic Kidney Disease in the Domestic Cat: A Theoretical Development Program

This work presents a theoretical development program for a single-administration adeno-associated virus (AAV) gene therapy designed to treat chronic kidney disease (CKD) in the domestic cat. The approach centers on the delivery of an affinity-detuned variant of feline Apoptosis Inhibitor of Macrophage (m-fAIM), enginee...

Genetic background determines allele-specific Pfkelch13-mediated artemisinin tolerance and persistence in Plasmodium falciparum

Findings reveal that a parasite’s genetic background acts as a strict evolutionary filter, uncoupling short-term tolerance from long-term persistence and establishing that genomic context determines allele-specific resistance trajectories.

The Pariza Decision Tree for Safety Assessment of Microbial Enzymes: A Silver Anniversary Update

This review critically updates the Pariza microbial enzyme safety evaluation decision tree, which has been used by industry since the turn of the century, to reflect current biotechnology tools and microbial safety assessment practices. The updated decision tree preserves the principle that enzyme safety depends primar...

Advocates and sceptics: Mapping consumer segments for gene-edited foods in New Zealand

Gene-editing technologies offer transformative potential for sustainable food production, yet acceptance by some consumers remains a concern. This study examines New Zealand consumers’ attitudes toward gene-edited foods and regulatory reform, applying the cognitive-affective personality system model to uncover the psyc...

Spatially guided in vivo single-cell functional genomics of postnatal heart

A high-resolution spatial and temporal atlas of the postnatal mouse heart is generated by integrating single-nucleus RNA sequencing with image-based spatial transcriptomics, revealing dynamic cellular interactions, niche-specific signaling and transcriptional programs guiding cardiomyocyte maturation.

Boosting Ligand-Mediated Hepatic mRNA Delivery and Gene Editing Through Coordinated Pairing of Polyethylene Glycol-Modified Lipids

It is discovered that incorporating TriGalNAc-lipid into LNPs containing DSPE-PEG350 increased hepatic luciferase expression 4–6-fold over TriGalNAc-conjugated or unconjugated DMG-PEG2000 LNPs, and 60-fold over TriGalNAc-conjugated or unconjugated DSPE-PEG2000 LNPs in vivo.

SANGHATI23/genomic-evidence-reliability: GES v1.0 - Springer Revision Reproducibility Freeze

Genomic Evidence Stability (GES) — Springer Revision Reproducibility Freeze This release freezes the reproducibility package supporting the revised GES chapter submitted to the edited volume Artificial Intelligence in Therapeutic Innovation. Included analyses January 2023 → January 2026 ClinVar temporal validation Froz...

xuzhougeng/wisp-science: v1.10.0: MCP

<!-- release-title: v1.10.0: MCP --> wisp-science v1.10.0 v1.10.0 brings scientific MCP tools into Wisp's native Rust runtime. The bundled catalog provides 23 connectors and 247 tools across roughly 80 upstream databases, with browsable connector introductions and tool parameters. This release also improves first-time...

SANGHATI23/genomic-evidence-reliability: GES v1.0 - Springer Revision Reproducibility Freeze

Genomic Evidence Stability (GES) — Springer Revision Reproducibility Freeze This release freezes the reproducibility package supporting the revised GES chapter submitted to the edited volume Artificial Intelligence in Therapeutic Innovation. Included analyses January 2023 → January 2026 ClinVar temporal validation Froz...