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

Researchers at the Broad Institute of MIT and Harvard have developed a gene-editing treatment for prion disease that extends lifespan by about 50 percent in a mouse model of the fatal ...

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 ...

Machine learning models reveal that histone marks are predictive of gene expression across human cell types and highlight important nuances between natural control and the effects of CRISPR-Cas9-based ...

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 ...

Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

By editing thousands of genes in mouse stem cells, the scientists identified a list of over 300 that are crucial for neural differentiation.

CRISPR technology has revolutionized biology, largely because of its simplicity compared to previous gene editing techniques. However, it still takes weeks to learn, design, perform, and analyze ...

On Tuesday, the team behind the plan to bring mammoth-like animals back to the tundra announced the creation of what it is calling wooly mice, which have long fur reminiscent of the woolly mammoth.

Data Demonstrate Potent in vivo Gene Editing of Hematopoietic Stem Cells (HSCs) in the Bone Marrow with Systemic Delivery in Preclinical Murine Models Using Fusogen Technology Broadens Application of ...