What is gene therapy and its application in medicine?

Premkumar Daivasikamani*

The principle of Gene therapy is to correct or modify the genetic material inside a cell to treat or cure a genetic disorder or cancer. It works by adding a normal working gene, which acts by silencing an altered gene or editing a broken gene so that the body can make healthy proteins. Gene therapy is based on genetic engineering or human genetic modification. Different approaches to gene therapy are: 1. Gene transfer: Where a healthy copy of the gene.2 replaces a faulty, disease-causing gene. Gene addition: A new or modified gene is introduced into the body to help the body fight off a disease. 3. Gene editing: Here, gene therapy inactivates or turns off a bad gene that is not working correctly. A broken or mutated gene is repaired by using specialized gene-editing tools. Tools target the main cause of conditions like inherited genetic disorders, some cancers, different neuromuscular diseases, and blood disorders instead of managing symptoms only. Somatic Gene Therapy acts by targeting modified body cells. The changes produced by gene therapy affect only the treated patient and are not passed to their children. Germ line Gene Therapy targets and changes egg, sperm, or embryo cells. These changes are heritable and passed on to their children. It is restricted or banned in many countries due to safety and ethics. CRISPR is a specific, highly precise tool used to edit genes by cutting and rewriting native DNA. CRISPR is nothing but an advanced method of gene therapy. A specific, precise tool called CRISPR edits genes by cutting and rewriting native DNA.

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Published on: Oct 21, 2026 Pages: 1-6

Full Text PDF Full Text HTML DOI: 10.17352/amgm.000016
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