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Gene Therapy – UPSC

Gene Therapy

Gene Therapy involves treating a genetic defect by introducing a functional gene or segment of DNA into cells. This functional gene, when inserted into an individual or embryo, serves to compensate for the non-functional gene that is present. By introducing this new DNA, the adverse effects of the mutated gene can be corrected. The concept of gene therapy originated in 1972, and it saw its first application in a therapeutic context in 1990 when a four-year-old girl received gene therapy to address adenosine deaminase (ADA) deficiency.

Types of Gene Therapy

Basically, there are two types of gene therapy.

Somatic Gene Therapy

This particular kind of gene therapy typically happens in the cells that make up our bodies, like skin or muscle cells. It’s all about helping just one person, replacing their damaged cells with healthy ones. What happens is that they introduce helpful genes into either the regular cells or the special cells in our bodies that can grow into different types of cells. People see this as the top choice for gene transfer because it’s really safe and effective.

Germline Gene Therapy

It occurs in the germline cells of the human body. Generally, this method is adopted to treat the genetic, disease causing-variations of genes which are passed from the parents to their children. The process involves introducing a healthy DNA into the cells responsible for producing reproductive cells, eggs or sperms. Germline gene therapy is not legal in many places as the risks outweigh the rewards.


  • Response from immune system: our immune system might not accept newly introduced gene
  • Mutation of undesired Gene and collateral effect
  • High cost for treatment of disease through gene transfer
  • Delivery of desired Gene to right place
  • Scientists can make mistakes and it can lead to occurrence of unwanted mutations
  • Possibility of Bio war
  • Virus is used as vector and it can show toxicity


  • It is used in the replacement of genes that cause medical ill-health
  • The method generally destroys the problem causing genes
  • It helps the body to fight against diseases by adding genes to the human body
  • This method is employed to treat diseases such as cancer, ADA deficiency, cystic fibrosis, etc.

    Read also: Principles of Biotechnology

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