Researchers have used a personalised base-editing gene therapy to successfully treat an infant with carbamoyl-phosphate ...

Positive preclinical POC data show that novel gene therapy, AXV-201, prevented obesity and metabolic disease in a monogenic ...

"That's because it breaks a gene, it inactivates it from doing what the cell needs it to do." It basically put a stop sign, called a stop codon, toward the beginning of the KEAP1 gene. Stop codons ...

The DNA is basically a book of recipes. Each recipe tells the cells in our bodies how to make specific proteins by combining ...

Researchers have known for quite some time that families can carry and pass on gene variants that raise your risk for cancers of the breast, ovaries, uterus, and colon, among others. More recently ...

She has the most common fatal genetic disease in the U.S., which afflicts 40,000 Americans. But her case is caused by a rare genetic mutation, so medications that work for 90% of people with ...