The FDA is considering approving the first gene-editing treatment targeting sickle cell disease. The treatment involves editing genes using CRISPR technology.
CNN interviews a young patient, Johnny Lubin, who underwent gene editing as part of a clinical trial. Born with sickle cell disease, Johnny faced chronic pain and hospitalizations. The new treatment, involving the precise editing of cells, potentially offers a cure for the disease by inducing fetal hemoglobin production.
Despite concerns about complications and costs, the treatment has shown positive results, with most patients, including Johnny, being free from pain crises for at least a year after treatment. Johnny's family celebrates his treatment day as his second birthday.
While uncertainties remain about the treatment's long-term effects and its wider usage, it signifies hope for patients with sickle cell disease.
Vocabulary:
• Approval: Formal permission or consent.
• Gene-editing: Modifying genes using specific technology.
• Crispr: A tool used for editing genes.
• Hemoglobin: A protein in red blood cells that carries oxygen.
• Clinical trial: Medical research involving volunteers to test treatments.
Source: CNN