CRISPR gene-editing is a revolutionary technology that acts like molecular scissors to make precise cuts in DNA. In the context of this therapy, it is used to disable the faulty gene that causes sickle cell disease and beta thalassemia.
2. How does the gene therapy process work?
The process involves extracting bone marrow stem cells from a patient, editing the genes in these cells in a laboratory to correct the mutation, and then re-infusing the modified cells back into the patient. This allows the body to start producing functional hemoglobin.
3. What is the main goal of this gene therapy?
The primary objective of the therapy is to enable the body to produce functional hemoglobin, which is crucial for transporting oxygen. This is particularly important for patients with sickle cell disease and beta thalassemia, who lack functional hemoglobin.
4. How effective has the gene therapy been in clinical trials?
Clinical trials have shown promising results. A significant number of patients with sickle cell disease and beta thalassemia experienced substantial improvements, with many no longer requiring regular blood transfusions.
5. What impact does this gene therapy have?
This gene therapy offers the potential for a permanent cure for patients with these blood disorders, significantly enhancing their quality of life and reducing the lifelong healthcare costs associated with managing these conditions.
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