Question

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Define the term gene therapy. Based on your definition, explain the reason gene therapy is only beneficial for some genetic disorders and not others.

Answer 1

Gene therapy: It is a collection of experimental technique, which was most predomonantly used to correct defective genes (responsible for disease devlopment) that has been diagnosed in a child/embryo. Correction of genetic defect involves delivery of a normal gene into the indivisual or embryo to take over the function of and compensate for 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 crucial for the immune system to function.this disorder is caused due to deletion of the gene for adenosine deaminase. The most common form of gene therapy involves inserting a normal gene to replace an abnormal gene. It is of two types Germ line gene therapy and Somatic cell line gene therapy.

Gene therapy work:

During the gene therapy a new designed genetic material was introduced into the cell for a beneficial protein making. In the case of mutated gene can only produce faulty or missing protein but in the process of gene therapy a normal copy of the gene was introduced and it can able to restore the function of the protein. So the gene therapy process is only beneficial for some genetic disorders when compared with other methods.

Benefits of gene therapy:

It has the ability to replace defective cells.

It can help eradicate diseases.

One great aspect about gene therapy that is noted by scientists and researchers is the remarkable therapeutic potential it promises.

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