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History of Gene Therapy Gene therapy is the use of genetic material- DNA or RNA- as a medicine. Classical gene therapy involves the introduction of functional genes, in the form of DNA, to replace mutated genes.This approach has been used to successfully treat a number of diseases including X-linked SCID, ADA-SCID, haemophilia B, lipoproteinlipase deficiency and Leber's congenital amaurosis with intensive research on-going for other inherited diseases. More recently scientists are looking at using gene technology to repair rather than replace mutated genes, silence overactive genes and to also provide our immune cells with the tools they need to recognise and kill cancer cells and infections.
What is Gene Therapy • Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient's cells instead of using drugs or surgery. • Gene therapy is when DNA is introduced into a patient to treat a genetic disease. The new DNA usually contains a functioning gene to correct the effects of a disease-causing mutation. • Gene therapy could be a way to fix a genetic problem at its source. By adding a corrected copy of a defective gene, gene therapy promises to help diseased tissues and organs work properly.
Needs of Gene Therapy • To overcome the disease. • Targeting the cell of diseased tissues. • Genes can be inserted into the cell of patients by direct and indirect routes, a& inserted the genes can integrate into the chromosomes.
Types of Gene Therapy Two Types of Gene Therapy • Germ line Gene Therapy. • Somatic gene Therapy.
Germ Line Gene Therapy • Therapeutic genes transferred into the germ cells. • It is heritable and passed on to later generations. • For safety, ethical and technical reasons, it is not being attempted at present. • Potential for offering a permanent therapeutic effect for all who inherit the target gene.
Somatic Gene Therapy Insertion of therapeutic gene into somatic cells like fibroblasts,myoblasts,epithelial cells, nervous cells,glial cells etc. This can correct the genetic defect in the patient. However,in somatic cell therapy, Transgene cannot be passed on to the siblings etc. Affected only the targeted cells in the patient, and it not passed to future generation.
Method Adopted of Gene Therapy • Ex-Vivo • In-Vivo
Ex Vivo Ex vivo, where cells are modified outside the body and then transplanted back in again.
In Vivo In vivo, which means interior where genes are changed in cells still in the body.
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