Liver cells are indispensable for research—for drug testing, to better understand diseases such as hepatitis, fatty liver, cirrhosis, or liver cancer and for development of future cell therapies.

Gene editing has emerged as a powerful approach for targeting the genetic causes of disease, yet delivering the editing machinery into the correct cells efficiently, safely, and at the scale needed ...

CRISPR functions as a programmable genetic memory system derived from bacterial immune defenses against viral infections. Guide RNA allows for rapid multiplexed targeting compared to older ...

Gene therapy has given sickle cell disease what medicine has long promised but rarely delivered: the possibility of a cure. Yet the slow uptake of Casgevy and Lyfgenia shows that even dazzling science ...

Cells contain a blueprint in the form of DNA that dictates what they can make. This blueprint is converted into a message (mRNA), which is then converted into a protein. Although DNA remains the same ...

Emerging gene editing platforms are revolutionizing precision medicine, enabling safe and effective genetic modifications for ...

A new gene-editing technique enables the correction of multiple genetic mutations simultaneously, transforming the prospects for millions living with complex inherited diseases, such as cystic ...

We tend to view ourselves and the complex cells that build us as a distinct branch of the tree of life from the compact, ...

Hundreds of genes have been linked to autism, yet the precise molecular and cellular mechanisms behind it remain largely unclear. A new study published in Nature, led by Gaia Novarino at the Institute ...