Sickle Cell Anemia is a genetic and hereditary disease of the blood. People with this disease have an irregular shape of red blood cells. The National Instutes of Health (NIH) has improved the development of a viral vector to be used for gene therapy for Sickle Cell Anemia. The disease is caused by a mutation of the Hemoglobin Beta chain gene, the mutation alters the shape of the chain, thus modifying the entire hemoglobin. The new vector developed by the NIH would succeed in inserting in the stem cells of the spinal cord the “correct” gene of the Beta chain of the hemoglobin, allowing the development of functioning blood cells. The researchers, who did tests on monkeys and mice, verified that this new vector was able to introduce an amount up to six times higher than the old vector of therapeutic beta-globin genes. The new vector has yet to be tested clinical on humans, NIH is working to speed up this and other genetic therapies, so as to reduce the negative impact of blood disorders.
If you want to learn more, we suggest reading the NIH article.