GENE THERAPY
Last Updated : 4/5/2010
Dr. Ira Shah
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What are genes?
Genes are biological units of heredity. Genes determine the hair and eye colour, as well as more subtle characteristics such as ability of the blood to carry oxygen or complex traits, physical strength etc.

What is

gene therapy

?

Gene therapy is an experimental medical intervention that involves modifying the genetic material of living cells to fight disease. It is still experimental. Goal of gene therapy is to supply cells with healthy copies of missing or altered genes. Gene therapy is the future therapy for incurable disease like cancer and genetic disorders.

Which is the first disease for which gene therapy is available?
The first disease approved for treatment with gene therapy was Adenosine deaminase deficiency (ADA). It is rare genetic disease in which the child has no immunity and usually dies by 2 years of age.

How is the healthy gene given?
The healthy gene is linked with a virus which is then injected into the body. The virus attaches to the various cells of the body and carries the gene with it. The genetic material then attaches to the cell's genetic material which starts to carry its own function.

What

risks

are associated with current gene therapy?

Viruses can usually infect more than one type of cell whereas gene needs to be inserted only in the cell in which that genetic material is absent. Another danger is that the new gene might be inserted in the wrong location in the cell, possibly causing cancer or other damage. Sometimes it may also change cell functions. However, scientists use precautions to identify and avoid these risks before the gene therapy can be put for general use.



Contributor Information and Disclosures

Dr. Ira Shah
Consultant Pediatrician, B.J.Wadia Hospital for Children, Mumbai, India


First Created : 5/1/2001

References

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