Define Gene Therapy. Can you explain it using an example of Adenosine deaminase (ADA) deficiency?
A collection of methods that helps the correction of gene deletions or gene defects is known as gene therapy. This method involves inserting a gene into an individual's cells and tissues to address a specific genetic disorder. This involves transferring the normal gene to an individual or embryo, replacing the defective mutant allele of the gene. It is used as a viral vector to attack the host cell and introduce the genetic material into the host. For example, adenosine deaminase (ADA) deficiency can be cured in some children through bone marrow transplantation, but gene therapy does not. Lymphocytes proliferate in culture and functional ADA, and cDNA is introduced into these lymphocytes. If so, these lymphocytes are transferred to the patient's body and the patient needs an injection of such genetically engineered lymphocytes.
