Gene Therapy
Gene therapy is a medical treatment that aims to replace or repair faulty genes. In order to do this, a functional gene copy is inserted into the patient's cells, thereby compensating for the non-functional gene. Gene therapy has the potential to treat a wide range of genetic disorders, including inherited diseases, as well as some cancers and viral infections.
Types of Gene Therapy
There are two main types of gene therapy: somatic gene therapy and germline gene therapy.
- Somatic gene therapy involves targeting the non-reproductive cells of the body, such as muscle or liver cells.
- Germline gene therapy targets the reproductive cells, such as sperm or egg cells. Germline gene therapy is controversial because any changes made to the genes in these cells would be passed on to future generations.
Methods of Delivering Functional Gene
There are several different methods of delivering the functional gene to the patient's cells.
- One common approach is to use a virus as a vector to carry the gene into the cells. The virus is modified so that it does not cause disease but can still deliver the gene.
- Another approach is to use a technique called CRISPR-Cas9 to edit the patient's own DNA and correct the faulty gene.
Challenges of Gene Therapy
While gene therapy has shown promise in treating genetic disorders, there are still many challenges to overcome.
- One major challenge is ensuring that the functional gene is inserted into the correct place in the patient's DNA, and that it does not disrupt any important genes or regulatory regions.
- Another challenge is ensuring that the functional gene is expressed at the correct levels, and that it does not cause any unintended side effects.
Despite these challenges, gene therapy has the potential to revolutionize the treatment of genetic disorders and improve the lives of millions of people around the world.
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