Question

wht is gene therapy​

Answer 1

$\huge\tt\red{A}\tt\pink{N}\tt\blue{S}\tt\green{W}\tt\purple{E}\tt\orange{R}$

a treatment in which the genes that are missing or not normal in your cells are replaced with normal genes

Answer 2

$\underline \mathscr \purple{Question \: : - }$

wht is gene therapy_??

$\underline \mathscr \purple{Answer \: : - }$

Gene Therapy

This is the name originally given to methods that aim to cure an inherited disease by providing the patient with a correct copy of the defective gene, Gene therapy has now been extended to include attempts to cure any disease by introduction of a cloned gene into the patient. If a person is born with a hereditary disease, then gene therapy can be a corrective therapy for such a disease.

Gene therapy is a collection of methods that allows correction of a gene defect that has been diagnosed in a child/embryo. Here genes are inserted into a person's cells and tissues to treat a disease. Correction of a genetic defect involves delivery of a normal gene into the individual or embryo to take over the function and compensate for the non-functional gene.

The first clinical gene therapy was given in 1990 to a four-year old girl with adenosine deaminase (ADA deficiency). This enzyme is crucial for the immune system to function because in its absence lymphocyte proliferation is inhibited. Without T cells, ADA-deficient children are wide open to the attacks of viruses and bacteria. The SCID (Severe Combined Immuno deficiency Disorder) is caused due to deletion of gene for adenosine deaminase.

In some children, ADA deficiency can be treated by bone marrow transplantation in others it can be treated by enzyme replacement therapy (ERT). In ERT, patient is given an intravenous injection of ADA or enzyme lacking in the content. But the problem with both approaches is that they are not completely curative.

As a first step towards gene therapy, lymphocytes from the blood of the patient are grown in a culture outside the body. A functional ADA CDNA (using a retroviral vector) is then introduced into these lymphocytes, which are subsequently returned to the patient. However, as these cells are not immortal, the patient requires periodic infusion of such genetically engineered lymphocytes. However, if the gene isolated from marrow cells producing ADA is introduced into cells at early embryonic stages, it could be a permanent cure.