Gene therapy for sickle cell disease

Subject :Science and Tech

Section: Health

Context:

Britain’s medicines regulator has authorized the world’s first gene therapy treatment for sickle cell disease.

About the new therapy:

Name of the therapy: Casgevy
Developed by: Vertex Pharmaceuticals (Europe) Ltd. and CRISPR Therapeutics.
Developed using: Gene editing tool CRISPR, which won its makers a Nobel prize in 2020.
Approved for: Patients with sickle cell disease and thalassemia who are 12 years old and over.
Earlier treatment: To date, bone marrow transplants, extremely arduous procedures that come with very unpleasant side effects, have been the only long-lasting treatment.

How Casgevy works?

It works by targeting the problematic gene in a patient’s bone marrow stem cells so that the body can make properly functioning hemoglobin.
Patients first receive a course of chemotherapy, before doctors take stem cells from the patient’s bone marrow and use genetic editing techniques in a laboratory to fix the gene.
The cells are then infused back into the patient for a permanent treatment.
Patients must be hospitalized at least twice — once for the collection of the stem cells and then to receive the altered cells.

About Sickle Cell Disease and Thalassemia:

Both sickle cell disease and thalassemia are caused by mistakes in the genes that carry hemoglobin, the protein in red blood cells that carry oxygen.
In people with sickle cell a genetic mutation causes the cells to become crescent-shaped, which can block blood flow and cause excruciating pain, organ damage, stroke and other problems.
It occurs more often among people from places where malaria is or was common, like Africa, Caribbean and India, and is also more common in certain ethnic groups, such as people of African, Middle Eastern and Indian descent. Scientists believe being a carrier of the sickle cell trait helps protect against severe malaria.
In people with thalassemia, the genetic mutation can cause severe anemia. Patients typically require blood transfusions every few weeks, and injections and medicines for their entire life. Thalassemia predominantly affects people of South Asian, Southeast Asian and Middle Eastern heritage.

Source of this article: AP