What is the revolutionary gene-editing treatment Adam received?

Adam received Casgevy (exa-cel), a one-time gene therapy utilizing CRISPR technology to treat the blood disorder thalassaemia.

How does the Casgevy gene therapy work for thalassaemia?

Casgevy works by editing faulty genes within a patient's own stem cells to prompt the production of healthy red blood cells.

What was Adam's condition before the gene therapy?

Before the gene therapy, Adam had beta-thalassaemia and relied on monthly blood transfusions since he was eight months old.

Home / Health / UK Child's Life Transformed by Gene Editing

UK Child's Life Transformed by Gene Editing

29 Jan

•

Summary

A boy received pioneering gene-editing therapy for thalassaemia.
The £1.65 million treatment uses CRISPR technology to edit faulty genes.
He no longer needs monthly blood transfusions after successful therapy.

UK Child's Life Transformed by Gene Editing

A 13-year-old boy from Oldham has had his life transformed by a groundbreaking gene-editing therapy, becoming one of the first in the UK to receive the treatment. Adam Rehman was born with beta-thalassaemia, a severe inherited blood disorder that previously necessitated monthly blood transfusions.

The one-time gene therapy, known as Casgevy (exa-cel), utilizes CRISPR gene-editing technology to correct faulty genes within the patient's own stem cells. This scientific breakthrough, recognized with a Nobel Prize, has now offered Adam a cure.

Adam received the specialized treatment in November 2024, a process involving the collection of his stem cells, gene editing in a lab, and reinfusion. This therapy provides a crucial option for patients who could benefit from a stem cell transplant but lack a suitable donor.