This technique is a targeted therapy and can be used on individuals as well as embryos. Gene therapy, combined with reduced-intensity conditioning, is a safe and effective treatment for SCID in patients with ADA deficiency. Note: Gene therapy is a treatment for a number of diseases such as cancers, certain viral infections, or some inherited disorders. Inserted gene either integrates into the chromosome of the host at different sites away from the mutated gene or replaces the defective gene and produces functional enzymes. For the repair of this effect, a retrovirus containing a functional wild-type ADA gene is injected into the parent's bone marrow ex-vivo and then again incorporated into the parent’s bone marrow. Severe Combined Immunodeficiency (SCID) is a rare genetic condition characterized by a lack of B- and T-lymphocytes, which form part of the adaptive immune system. In SCID, a gene known as ADA gene that codes for the enzyme adenosine deaminase is mutated. There are certain challenges in this method as a new gene is inserted into the body, the immune system of our body can reject the gene due to the action of our body cells on the foreign target. This method is adopted to treat the genetic disorder causing variations of genes which are passed on from parents to their offsprings. This process involves transferring healthy DNA into the cells responsible for producing reproductive cells i.e. Germline Gene Therapy- It usually occurs in the germline cells (sex cells) of the body. SCID comprises of a group of inherited defects that results in impaired T cell development or function. In this therapy, therapeutic genes or desired genes are transferred into the somatic cells or the stem cells of the body. This condition causes an incredibly weak immune. Somatic Gene Therapy- It usually occurs in the somatic cells (vegetative cells) of the body. Severe combined immunodeficiency disease (SCID) is a very rare genetic disorder that affects about 1 in every 100,000 births in the United States. There are two types of gene therapy depending upon the type of cells involved: The disease is also known as the boy in the bubble syndrome because living in a normal environment can be fatal to a child who has it. As a result, the child is unable to fight off even mild infections. It causes a child to have a very weak immune system. First gene therapy was successfully done in the year 1989. SCID is a very rare disease that can be deadly. Gene therapy is an artificial method that introduces DNA into the cells of the human body. Adenosine deaminase (ADA) deficiency, sometimes called ADA SCID, is caused by a mutation in the gene that encodes the protein called adenosine deaminase. It involves the replacement of defective or faulty genes with healthy ones in order to treat genetic disorders. Gene therapy or gene transfer therapy is an experimental technique that uses genes to treat a disease. Gene therapy is a new technique for the treatment of the disease at the genetic level. Hint: Gene therapy technique is based on recombinant DNA technology.
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