Sickle cell anemia patient ‘cured’ by using gene therapy

Sickle cell anemia is a deadly disease. People who are diagnosed with this disease are usually between 40 and 60. But that will cause those a lifetime of pain, where the hemoglobin stops up blood flow and deprives tissues of oxygen, along with the consequences like organ damage. Researchers have been searching for a cure from decades ago, and now at least one patient, they seem to have found a very promising treatment.

A French teenager having sickle cell anemia had undergone a gene therapy treatment that was supposed to rescue the red blood cells from sickling. In the paper published recently, the researchers have found that his red blood cells have become normal-shaped hemoglobin. There was no need for blood transfusion, which many sickle cell patients receive to reduce complications from the disease, after three months of his treatment.

Bluebird Bio, a biotech company based in Massachusetts, had sponsored the clinical trial. The gene therapy has not worked quite as well in some of those other patients. Scientists say that they are adjusting the therapy accordingly. It is also possible that the boy may eventually experience some blood flow blockages again in the future.

 Sickle cell anemia is an inherited genetic disease that is caused by a mutation of a single letter in a person’s genetic code. This single-letter mutation makes it a promising candidate for cutting edge technologies, like the gene-editing technique CRISPR-Cas9.

Currently, the only long-term treatment for sickle cell disease is a bone marrow transplant, a high-risk, difficult procedure that many patients are not even eligible for. Pain and other side-effects are treated with blood transfusions for temporary relief. New technologies offer the hope of a solution that could provide long-term relief and allow patients to live some semblance of a healthy life.

Scientists have taken bone marrow stem cells from the boy and fed them corrected versions of a gene that codes for beta-globin, a protein that helps produce normal hemoglobin. The stem cells will interfere with the boy’s faulty proteins and allow his red blood cells to function normally.

They continued the transfusions until the transplanted cells began to produce normal-shaped hemoglobin.

For ages, gene therapies have been using as a cure for everything. But so far, successes have been infrequent, and often for very rare diseases. If we are lucky, the benefit of this technology will help thousands of people worldwide.

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