Gene Therapy

Dr. Ira Shah
Consultant Pediatrician, B.J.Wadia Hospital for Children, Mumbai, India
First Created: 05/01/2001 


What are genes?

Genes are biological units of heredity. Genes determine the hair and eye color, as well as more subtle characteristics such as the 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. The goal of gene therapy is to supply cells with healthy copies of missing or altered genes. Gene therapy is the future therapy for an 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 a 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 gene therapy can be put for general use.

Gene Therapy Gene Therapy 2001-05-01
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