An in vivo base editing approach targeting the PRNP gene led to a 52% increase in the lifespan of mouse models inoculated with the most common sporadic and genetic types of human pathogenic prion ...

The TP53 gene is a gene that is mutated in many cancers. It is the most common gene mutation found in cancer cells. A tumor-suppressor gene, TP53 codes for a protein that inhibits the development and ...

We identified 82 cases with pathogenic or likely pathogenic SNVs and 4 cases with pathogenic CNVs, of which one case with both SNV and CNV. The most frequently detected gene was PRRT2, present in 10.0 ...

FXS is caused by a mutation in FMR1, a gene which modulates a number of systems, including the endocannabinoid system, and most critically, codes for a protein called FMRP. The FMR1 mutation ...

The first human trial of insulin-producing cells that have been gene-edited to evade immune attack is a success so far. The cells have survived and produced insulin for a month after being ...

This complex process, known as gene expression, determines which proteins we make and whether we make them in the correct amounts, crucial work given that proteins play a role in virtually every a ...

A ba­by boy born with a de­bil­i­tat­ing ge­net­ic dis­ease will like­ly no longer re­quire a liv­er trans­plant to sta­bi­lize his con­di­tion, thanks to a first … ...

Summary: Researchers have developed an AI model that accurately predicts gene activity in any human cell, providing insights into cellular functions and disease mechanisms. Trained on data from over 1 ...

The same is now possible in gene editing. Rather than employ separate tools for editing genes and regulating their expression, these distinct goals can now be combined into a single tool that can ...

Participants must have a confirmed variant in the SCN1A gene not linked to a gain of function. Dravet syndrome is a debilitating condition with high unmet medical needs, characterized by frequent ...

Some genes are beneficial and enhance longevity. For example, the gene that helps a person metabolize cholesterol would reduce a person's risk of heart disease. Some gene mutations are inherited.