Question

Describe gene therapy?  Choose any human condition that is currently being treated with gene therapy. Identify and describe the genetic deficiency that the strategy is intended to address. Describe the proposed gene therapy procedure. Identify successes, failures, or observed adverse consequences that have been associated with this condition in gene therapy in the past or currently. 100 words minimum

Answer 1

Gene therapy is the treatment of diseases using genes. The damaged or mutant gene is replaced with working gene in this novel treatment. It can be used to treat hereditary diseases. For the introduction of this working gene into the cell, a carrier is genetically engineered. This carrier is called a vector. Some viruses such as retrovirus are used as vectors.

Hemophilia is currently being treated with gene therapy. Hemophilia is the condition of non existence of clotting of blood in the body. That is the patient suffers from external as well as internal bleeding because his blood won't clot. This is due to the fault in the genes which are necessary for the production of clotting factors.Clotting factors are the proteins in blood that help our bodies to stop bleeding. So, gene therapy would address this by delivering a functional version of the non-working gene into a cell, instructing it to produce the missing clotting factor. Using a vector, this functional gene is delivered. The vectors are delivered into the liver cells. And thus, the functional gene will instruct the liver cells to produce clotting factors to help prevent the bleeding. But there was a problem because, the genetically corrected liver cells were identified as foreign bodies by the immune system and were rejected. So, we have to couple immune-suppression along with the gene therapy in the future for successful results.