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

Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic ...

FDA Commissioner Marty Makary says the agency wants to remove barriers for patients suffering from rare genetic conditions.

Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders Blok–Campeau syndrome caused by mutations in the CHD3 gene. A specialized ...

The framework, first introduced by FDA Commissioner Marty Makary and Center for Biologics Evaluation and Research head Vinay ...

Gene editing has enormous potential to help feed the world’s growing population, but it’s currently difficult, time-consuming ...

A refined base-pair editor limits unintended neighboring DNA edits while maintaining strong correction of disease-causing mutations. The technology could advance treatments for cystic fibrosis and ...

Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic "base pairs" to a new level ...

As Uganda rolls out mandatory nationwide screening of newborns for sickle cell disease this month, a gene therapy celebrated in the United States and Europe remains financially out of reach for most ...

Scientists have long sought to understand why some plants are fragrant powerhouses while others remain subtle. Now, a ...

Vector-borne diseases (VBDs) represent a critical challenge in infectious disease control due to their complex transmission dynamics, diverse vector ...

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