Scientists have identified how specific genetic changes function in cells to influence disease risk and other human health ...

Gene therapy has been successfully used to treat a number of diseases, including immune deficiencies, hereditary blindness, ...

"Gene editing takes centre stage in FDA’s new rare disease approval pathway" was originally created and published by ...

Genetic changes can signal evidence of disease, but pinpointing which genes and what’s changed can be difficult. But in a study of traits that offer clues of a person’s cardiovascular health—such as ...

Genetic research is finally starting to explain how DNA risk scores translate into real disease, not just in single genes but across entire biological networks. Instead of treating each variant as an ...

Nature and nurture both determine how likely you are to develop a particular disease. Hiroshi Watanabe/DigitalVision via Getty Images Sitting in my doctor’s examination room, I was surprised when she ...

Researchers at the Weill Institute for Cell and Molecular Biology have uncovered new evidence that two major types of gene-controlling DNA sequences, promoters and enhancers, operate with a shared ...

The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are ...

CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...

For more than six decades, biomarker-based newborn screening has played a pivotal role in reducing infant mortality and ...

In many countries, newborn screening programmes test babies for certain inherited conditions shortly after birth.

With 300 million people living with rare diseases, experts say new gene and cell therapies are giving families in Wisconsin hope for lasting treatment options.