Researchers at the University of Hawaiʻi at Mānoa have developed new mouse models to study a key male fertility gene, ...

Genetic disorders occur due to alterations in the primary genetic material—deoxyribonucleic acid (DNA)—of an organism.

SCD gene therapy approaches using CRISPR, gene therapy, and base editing show different stem cell outcomes in a mouse study.

Markus Terrey, a neuroscientist at The Jackson Laboratory’s Rare Disease Translational Center, led the development of the new AHC mouse models—breakthrough tools that are helping researchers ...

Therapeutic Potential of CRISPR–Cas3 Genome-Editing System for Transthyretin Amyloidosis. Credit: Institute of Medical ...

Infants born with alternating hemiplegia of childhood (AHC) begin experiencing episodes of paralysis and seizures by the time they are a few months old, followed by developmental delays and ...

Toward the goal of efficient editing of farm animals for genetic improvements, this study explores an upgraded form of nuclease PE (uPEn). Based on an initial demonstration of its success in editing ...

A gene-editing delivery system developed by UT Southwestern Medical Center researchers simultaneously targeted the liver and lungs of a preclinical model of a rare genetic disease known as alpha-1 ...