Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders ...

New Program Designed as Platform-based Approach for Direct Correction of Mutations Causing PKU; Investigational New Drug (IND) Filing for ...

Beam is advancing BEAM-304 using an approach in which multiple mutation-specific base editors are developed within a single clinical program.

A refined base-pair editor limits unintended neighboring DNA edits while maintaining strong correction of disease-causing ...

Scientists at the Icahn School of Medicine at Mount Sinai have developed a novel artificial intelligence tool that not only identifies disease-causing genetic mutations but also predicts the type of ...

A seminal study has uncovered a new genetic cause of neurodevelopmental disorders (NDDs). The discovery offers both closure and hope to potentially thousands of families worldwide who have long been ...

An Ice Age double burial in Italy has yielded a stunning genetic revelation. DNA from a mother and daughter who lived over 12,000 years ago shows that the younger had a rare inherited growth disorder, ...

TSRA-196 is designed to precisely correct the genetic mutation underlying AATD, with the goal of restoring production of functional alpha-1 antitrypsin (AAT) protein through a one-time, durable ...

Genetic inheritance may sound straightforward: One gene causes one trait or a specific illness. When doctors use genetics, it’s usually to try to identify a disease-causing gene to help guide ...