Gene can therapy can be referred to asa)pre - clinical testing for inh...
Gene therapy is a collection of methods, that allows correction of gene defect that has been diagnosed in a child/embryo. Correcting of a genetic defect involves delivery of a normal gene into the individual or embryo to take over the function of and compensate for the non-functional gene.
View all questions of this testGene can therapy can be referred to asa)pre - clinical testing for inh...
Treatment of Diseases caused by Genetic Defect
Gene therapy is a medical treatment that involves altering the genes inside a person's cells to treat or cure disease. It can be used to treat diseases caused by genetic defects, such as cystic fibrosis, sickle cell anemia, and certain types of cancer.
How it works?
Gene therapy works by introducing new, healthy genes into a person's cells to replace or supplement the defective ones. There are two main types of gene therapy:
1. In vivo gene therapy: This involves delivering the therapeutic genes directly into the patient's body. This can be done using a viral vector, which is a virus that has been modified to carry the therapeutic genes.
2. Ex vivo gene therapy: This involves removing cells from the patient's body, modifying them outside the body, and then returning them to the patient's body. This can be done using a variety of techniques, including viral vectors and CRISPR-Cas9 gene editing.
Benefits of Gene Therapy
The benefits of gene therapy are significant. It has the potential to cure or treat diseases that were previously considered incurable, and it can improve the quality of life for patients with genetic diseases. For example, gene therapy has been shown to:
- Cure severe combined immunodeficiency (SCID), also known as "bubble boy" disease
- Improve vision in patients with a rare form of blindness
- Treat certain types of cancer, such as leukemia and lymphoma
Conclusion
In conclusion, gene therapy is a promising medical treatment that has the potential to cure or treat a wide range of diseases caused by genetic defects. While there are still many challenges to overcome, such as the risk of unintended consequences and the high cost of treatment, the future of gene therapy looks bright.