Gene Therapy
The insertion of working copies of a gene into the cells of a person with a genetic disorder in an attempt to correct the disorder
Gene therapy is a biomedical approach that involves the delivery of genetic material into cells or tissues of an individual to treat or prevent disease. Essentially, it entails introducing a functional copy of a missing or faulty gene or modifying the expression of an existing gene to address a particular medical condition.
There are two main types of gene therapy: somatic cell therapy and germ line therapy. Somatic cell therapy targets non-reproductive cells of the body and does not affect the individual’s offspring, while germ line therapy targets reproductive cells and can lead to permanent changes that can be passed on to the next generation.
There are several methods of gene therapy delivery, including viral vectors, non-viral vectors, and genome editing. Viral vectors are currently the most efficient and popular method of gene delivery as they can effectively introduce foreign genetic material into cells. Non-viral vectors, such as plasmids, offer a safer option but are less efficient. Genome editing is an emerging technology that allows for precise modification or correction of genes at specific genetic loci.
Gene therapy has shown great promise in the treatment of a wide range of medical conditions, including genetic disorders, cancer, and infectious diseases. However, there are still many challenges to overcome, including efficient gene delivery, immune response to viral vectors, and ethical considerations.
Overall, gene therapy represents an exciting frontier in modern medicine with the potential to revolutionize the way we treat and prevent disease.
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