About Gene Therapy
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What is gene therapy?
Gene therapy is a treatment in which genes are used to treat disorders or diseases.
What is used in gene therapy treatment?Gene therapy treatment uses the genetic material, DNA, as the means of treatment. DNA or deoxyribonucleic acid is the very long molecule that encodes the genetic information.
What are genes?Genes are made up of DNA which carries information needed to make body proteins. The proteins are the building blocks of the body. There are about 100,000 to 150,000 genes present in humans.
How does gene therapy work?Gene therapy alters, replaces or supplements the abnormal or absent gene, whose absence or abnormality is responsible for the disorder. Normal DNA is isolated and then packed and transferred to the cell by a vehicle called as a vector.
What are the diseases that can be treated with gene therapy?Gene therapy can be used to treat various disorders such as cystic fibrosis, Alzheimer’s disease, arthritis, cancer, AIDS, cardiovascular diseases, amyotrophic lateral sclerosis.
Has gene therapy been successfully used in humans?Successful gene therapy was performed in 1990 to treat 4 yrs old child suffering from adenosine deaminase deficiency (ADA), which is a rare genetic disorder. Many studies are going on to find the effect of gene therapy on other disorders. They are more successful in disorder caused by single gene defect or due to gene interaction with the environment.
How genes are made to enter the body cells?Genes are introduced into the cells with the help of a carrier called as a vector. Mostly, these are viruses which enter the cells and transfer the genetic material.
What are the uses of gene therapy?- Gene therapy can help in boosting the immune response.
- Gene therapy can help taking genes into the cancer cells and make them more sensitive to particular treatments. Hence, they are helpful in chemotherapy and radiotherapy.
- Gene therapy can deliver genes into the cancer cells. This makes the cells to change drugs from an inactive form to an active form and hence kill the cancer cells.
- Gene therapy can help in cancer cells. Cell die when they are destroyed beyond repair by a process called apoptosis also called as programmed cell death. In cancer, this process is blocked, hence, cells don’t die when they are supposed to. Gene therapy can reverse this blockage and can make cancer cells to die.
Risks : Immune system may respond leading to inflammation due to the inserted genes, Virus vector may target genes other that intended ones, The activation of inserted gene may add to further health problems, The inserted genes may be placed in a wrong place, Virus vector may lead to further health problems
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