Question:

The first clinical gene therapy was given for treating

Updated On: Jul 29, 2022
  • diabetes mellitus
  • chicken pox
  • rheumatoid arthritis
  • adenosine deaminase deficiency.
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The Correct Option is D

Solution and Explanation

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 of and compensate for the non-functional gene. The first clinical gene therapy was given in 1990 to a 4 - year old girl with adenosine deaminase (ADA) deficiency. This enzyme is very important for the immune system to function. SCID is caused due to defect in the gene for the enzyme adenosine deaminase. In some children ADA deficiency can be cured by bone marrow transplantation. Here, the isolated gene from bone marrow cells producing ADA is introduced into cells at early embryonic stages; it can be a permanent cure.
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Concepts Used:

Applications of Biotechnology

Biotechnology is the technology that utilizes biological systems, living organisms, or parts of this to develop or create different products.

For example, brewing and baking bread fall within the concept of biotechnology (use of yeast (= living organism) to produce the desired product).

Agriculture

Alternative to conventional farming: A possible solution is the use of genetically modified crops which is an alternative path to conventional farming.

Medicine 

In the area of healthcare, recombinant DNA technological processes have made an immense impact which enables the mass production of safe and more effective therapeutic drugs.

Gene Therapy 

A collection of methods that allows the correction of a gene defect that has been diagnosed in a child/embryo is called Gene Therapy.

CRISPR technology

For editing genomes, CRISPR is a simple yet powerful tool which allows researchers to easily alter DNA sequences and modify gene function.