Question

Gene therapy is an attempt to correct a genetic defect by providing a normal gene into the individual. By this the normal function can be restored. An alternate method would be to provide the gene product (protein/enzyme) known as enzyme replacement therapy, which would also restore the function. Which in your opinion is a better option? Give reason for your answer.

Answer 1

Gene therapy :

Gene therapy is the collection of methods in which a defective gene present in the embryo or child is corrected by replacing it with a normal gene to compensate for the malfunctioning of the defective gene.

Enzyme replacement therapy :

Enzyme replacement therapy involves the replacement of non-functional enzymes produced from a defective gene with the functional enzymes.

Gene therapy is better than enzyme replacement therapy:

Gene therapy is better than enzyme replacement therapy due to the following reasons:

• When a gene is incorporated in the place of a faulty gene, it will take care of the synthesis of the enzyme.
• This method is much more simpler than utilizing the same gene to synthesize the enzyme outside the body,followed by its purification and further administering it.
• The enzyme replacement therapy requires periodic administering of the enzymes.
• But in gene therapy the enzyme is continuously produced in the body as long as the cells carrying the gene are viable.
• If the gene therapy is carried out at an early embryonic stage, the cure is permanent.