It’s a new way to create “bi-paternal” mice that can survive to adulthood—but human applications are still a long way off.

The UK’s Medicines and Healthcare Regulatory Agency (MHRA) approved Casgevy for sickle cell disease in 2023, followed by ...

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

Around 1,700 people could be eligible for the one-time treatment, which is seen as a less risky alternative to a donor stem ...

Researchers use CRISPR to overcome seemingly insurmontable reproductive barriers — but that doesn't mean this could work for ...

Experts hailed exciting news for some people with sickle cell disease in England as the health watchdog decided to grant access to gene-editing therapy on the NHS.

January 31, 2025 expert reaction to NICE final draft guidance on exagamglogene autotemcel (exa-cel) for severe sickle cell disease . Scientists comment on final draft guidance fro ...

Base editing utilizes enzymes to modify single amino acids at the most foundational unit of DNA, called a base.

The Exa-cel gene therapy treatment is at the forefront of medical technology. It takes the person’s DNA and “edits” out the faulty sickle gene. It’s expensive, too. According to the manufacturer, ...

The therapy from Vertex Pharmaceuticals and CRISPR Therapeutics will cost the ... The therapy involves taking stem cells out of a patient's bone marrow and editing a gene in the cells in a ...

Casgevy, also known as exa-cel, was the first treatment to be licensed using gene-editing tool Crispr, which earned its ...