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What is Gene Therapy? Gene therapy is an experimental technique that uses genes to treat or prevent disease by adding a corrected copy of a defective gene which will help diseased tissues and organs work properly again. It is different from traditional drug-based approaches, because not only does it treat the symptoms, but it also modifies the underlying genetic problems.

The Two Types of Gene Therapy Depending on the cells, gene therapy falls into two groups, somatic and Germline gene therapy Somatic Gene Therapy: Somatic gene therapy is the transfer of a section of DNA to any cell of the body that DOESN’T produce sperm or eggs and doesn’t get passed on to the patient’s child. Germline Gene Therapy Germline gene therapy is the transfer of a section of DNA to cells that DO produce eggs or sperm. With this type of gene therapy, the effects will be passed onto the patient’s children and other generations.

How it Works Most commonly, gene therapy uses a vector that delivers the gene such as a virus, to the cells where it's needed. Once its inside, the cell's gene-reading machinery uses the information in the gene to build RNA and protein molecules and then the proteins can then carry out their job in the cells.

Types of Vectors Used Retroviruses: Virus that uses RNA as its genetic material Adenovirus: Any of a group of DNA viruses first discovered in adenoid tissue Adeno-associated viruses: Adeno-associated virus is a small virus which infects humans and some other primate species. AAV is not currently known to cause disease Herpes Simplex Viruses: Common viral infection

Gene Therapy Time Line 1960 – 1970: The concept of gene therapy arose, however, research is still being done on the long-term data of the safety of this therapy. 1972: Theodore Friedmann authored a paper in Science titled "Gene therapy for human genetic disease?" Where they proposed that  good DNA  could be used to replace the defective DNA in those who suffer from genetic defects Theodore Friedmann

(Continued) 1984: A retrovirus vector system was designed that could efficiently insert foreign genes into mammalian chromosomes. 1990: On September 14, 1990, the first approved gene therapy procedure was performed on a four-year old patient who was born with a rare genetic disease. In her procedure, doctors removed white blood cells from her body and let the cells grow in the lab. Then they Inserted the missing gene into the cells, and then infused the genetically modified blood cells back into the patient's bloodstream.

Current Applications of Gene Therapy Cystic fibrosis is one of many diseases that can drastically alter the symptoms through the process of gene therapy. It is a heredity disorder that causes congestion in the lungs, but also damages parts of the digestive system. Gene therapy would allow you to insert the healthy version of the gene, which would allow for the cells in the lungs to make the correct protein that would no the produced otherwise.

Pros and Cons Pros Cons With reproductive cells being targeted, diseases that are transmitted by parents could be eradicated in the future.  Genetic disorders such as Alzheimer’s, Parkinson’s, and Cystic fibrosis are non-curable, but with gene therapy alone, we could replace the defective cells causing these diseases.   The capabilities of gene therapy are endless Most people do not understand the safety risks that go with gene therapy. Sometimes other genetic disorders can come about if the process goes wrong. If this technology is used repeatedly, we could start to see damage to our whole gene pool. The ethical issues around gene therapy are usually about the fact that we should not alter the genes that we are born with.

How its being used Even though gene therapy has been slow to reach patients, its still being used to clear up certain genetic defects. This includes things such as: Immune deficiencies Hereditary blindness Hemophilia Blood disease Cancer Fat metabolism disorder

Work Cited Page http://www.yourgenome.org/facts/what-is-gene-therapy, http://learn.genetics.utah.edu/content/genetherapy/gtintro/, http://cisncancer.org/research/new_treatments/gene_therapy/how_it_works.html, http://www.genetherapynet.com/patient-information.html