🔬CRISPR is a powerful gene editing tool that is revolutionizing medicine.
💉Casgevy is the first CRISPR-based gene therapy approved for treating sickle cell disease and β-thalassemia.
📝Lyfgenia uses an older gene editing technology called a lentiviral vector to treat sickle cell disease.
⏰Both treatments require a complex process, including chemotherapy and stem cell transplantation.
💰The high cost of these gene therapies limits access for many patients.
What is CRISPR?
—CRISPR is a gene editing tool that allows scientists to modify specific genes by targeting and cutting DNA.
What is Casgevy?
—Casgevy is a CRISPR-based gene therapy approved for treating blood disorders like sickle cell disease and β-thalassemia.
How does Lyfgenia work?
—Lyfgenia uses a lentiviral vector to deliver an anti-sickling version of hemoglobin to the patient's blood stem cells.
What is the process for receiving these treatments?
—The process involves regular blood transfusions, chemotherapy, stem cell transplantation, and recovery in a treatment center.
Why is access to these treatments limited?
—The high cost of gene therapies like Casgevy and Lyfgenia make them inaccessible for many patients.
00:00CRISPR, a powerful gene-editing tool, is being used to treat blood disorders like sickle cell disease.
03:42Casgevy is the first approved CRISPR-based gene therapy for sickle cell disease and β-thalassemia.
05:54Lyfgenia uses a lentiviral vector to treat sickle cell disease.
09:16Both treatments require a complex process involving chemotherapy and stem cell transplantation.
11:51The high cost of these treatments limits access for many patients.
