Sickle cell anaemia: gene therapy insights for blood disorders

Innovative gene therapy methods are revolutionizing the treatment approach of blood disorders like sickle cell anaemia, according to the latest research conducted by experts at Michigan Medicine, the Children’s Hospital of Philadelphia, and Penn Medicine. Current treatment requires aggressive chemotherapy and bone marrow transplants, but about 30% of patients do not have a suitable immune system match and those who do are at risk of the donor’s immune system attacking the recipient, a condition known as graft-versus-host disease. Gene therapy addresses this by rectifying genetic mutations within the patient’s cells through a process involving transplantation of the corrected cells after chemotherapy.

The latest research indicates that blood stem cells can be genetically modified directly within the bone marrow in a single procedure. Dr. Michael Triebwasser, a clinical instructor in Pediatric Hematology and Oncology and a co-first author of the study, believes this advancement could rectify disease-causing mutations such as those responsible for sickle cell anaemia in about 7.5 million people worldwide. Furthermore, this procedure reduces the need for stem cell harvesting and external treatment, potentially lowering treatment costs and widening patient access to crucial gene therapies.