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New hope for sickle-cell patients
News: With advances in gene therapy the prospects of curing sickle-cell disease seems applealing.
Facts:
- Sickle cell anemia is an inherited form of anemia — a condition in which there aren’t enough healthy red blood cells to carry adequate oxygen throughout the body.
- Normally, red blood cells are flexible and round, moving easily through the blood vessels. In sickle cell anemia, the red blood cells become rigid and sticky and are shaped like sickles or crescent moons.
- The misshapen cells don’t survive long in the blood — 10 to 20 days, compared to the usual 120 days. Patients may be severely anaemic and prone to infections.
- Children usually return to normal between crises, but teenagers and adults may suffer chronic pain.
Cure of the disease:
- At the moment, the only remedy for sickle-cell disease is a dangerous and expensive bone marrow transplant, an option rarely used.
- Scientists are researching on treating the disease through a gene editing technique and clinical trials have already shown reduced signs of disease.
- In the new trials, immature blood cells i.e. stem cells are removed from the bone marrow. The stem cells are genetically modified, and then infused back into the patient’s bloodstream.
- The goal is for the modified cells to take up residence in the bone marrow and form healthy red blood cells.
- An effective treatment can benefit a large number of poor sub-Saharan African people, a region where the disease is most prevalent.
