Is gene therapy available for thalassemia?
Called Zynteglo, the gene therapy is aimed at treating people with beta-thalassemia who are over 12 years of age and have any but the most severe form of the condition. Patients with beta-thalassemia often depend on receiving blood transfusions for their whole lives.
What current research is being done to develop a cure for thalassemia?
Scientists are working to develop a gene therapy that may offer a cure for thalassemia. Such a treatment might involve inserting a normal beta globin gene (the gene that is abnormal in this disease) into the patient’s stem cells, the immature bone marrow cells that are the precursors of all other cells in the blood.
What is gene therapy in thalassemia?
How gene therapy for beta thalassemia works. Blood stem cells are collected from a patient and genetically edited outside the body to block the expression of the BCL11A gene, which normally shuts off the production of fetal hemoglobin shortly after birth.
What is the future of thalassemia?
Future treatments for beta-thalassemia will depend on the benefit/risk/cost ratios for conventional transfusion and iron chelation [70], γ-globin induction [186], allogeneic bone marrow transplantation [187] and gene therapy [170], and the availability of these treatments in the low-income countries in which most β- …
Has sickle cell been cured?
It leads to chronic pain, organ failure, and early death. Currently, the only established method to cure sickle cell disease is a bone marrow transplant.
Has CRISPR been used on humans?
The first trial of a CRISPR-based therapy to treat inherited blindness. Doctors performing eye surgery. In a world first, CRISPR, the powerful gene-editing tool that can cut and paste DNA, has been used inside the human body for the first time.
Can thalassemia be cure by bone marrow transplantation?
The only radical cure for homozygous thalassemia is to transplant bone marrow from an HLA-identical donor who is normal or heterozygous for thalassemia, which is capable of producing and maintaining a normal hemoglobin level in the recipient.
What’s the cure for thalassemia?
Nutritional supplements, in the form of folic acid supplements, and monitoring of B12 levels are important, as these nutrients are key components to making healthy blood cells. Bone marrow and stem cell transplant from a compatible related donor is the only treatment that can cure thalassemia.
Is there any natural treatment for thalassemia?
Folate supplements are given to those with thalassemia major. This naturally occurring B vitamin is now available in a synthesized form as folic acid. Folate helps build red blood cells and is involved in the production of DNA.
What is thalassemia trait?
Thalassemia Trait. Thalassemia is a genetic disease. This means that a person can only get thalassemia disease or trait by inheriting the genes for thalassemia from their parents. Genes determine what we look like, such as hair color, and are also responsible for many diseases.
How is thalassemia inherited?
Thalassemia is inherited in an autosomal recessive manner. The genes are present on the chromosomes for expressing the globin, which is important in formation of hemoglobin. Any mutation of these genes may cause thalassemia.
What is gene and stem cell therapy?
Gene therapy and stem cell therapy are novel therapeutic techniques developed by researchers through advanced experiments. Gene therapy can be defined as a technique which introduces genes or genetic materials into patients to correct or replace abnormal or mutated genes that cause genetic diseases.