Gene Therapy

Gene Therapy is a medical procedure that involves the introduction, removal, or alteration of genetic material within a patient's cells to treat or prevent disease. Gene therapy is considered a form of biotechnology and is a rapidly evolving field in medicine.

Overview

Gene therapy works by replacing a faulty disease-causing gene with a working version, or by introducing a new gene to help fight a disease. This is usually done using a carrier, often a virus, to deliver the gene to the cells. This is known as a vector. The vector may be injected directly into a specific tissue in the body, where it is taken up by individual cells. Alternately, a sample of the patient's cells can be removed and exposed to the vector in a laboratory setting. The cells containing the vector are then returned to the patient.

Types of Gene Therapy

There are two main types of gene therapy: Somatic gene therapy and Germline gene therapy. Somatic gene therapy involves the manipulation of gene expression in cells that will be corrective to the patient but not inherited to the next generation. In contrast, germline gene therapy involves the genetic modification of germ cells that pass on changes to the next generation.

Applications

Gene therapy has potential applications in treating a variety of diseases such as cancer, genetic disorders, and certain viral infections. It is currently being studied for the treatment of diseases such as Huntington's disease, Parkinson's disease, and Hemophilia.

Risks and Challenges

Despite its potential, gene therapy poses significant risks and challenges. These include immune reactions, inflammation, gene control and targeting issues, and the possibility of causing a new disease.

See Also

• Genetic Engineering
• Biotechnology
• Genomics
• Genetic Disorders

References

Gene Therapy Resources
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