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 ...

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...

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 ...

Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

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 ...

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

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

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.