Chapter # 6: Gene Therapy FAQ

Chapter #6:

Gene Therapy FAQ

1.      What is gene therapy?

Gene Therapy is a way to treat diseases by correcting the faulty gene. A new functional gene is replaced by the faulty or non-functional one.

2.      How many types of gene therapy are there?

There are two types of gene therapy, somatic cell gene therapy, where a normal gene introduced to the body cells like skin, blood, and bone marrow. There is also germline gene therapy, where the new functional gene is introduced to germ cells (egg and sperms).

3.      How does gene therapy work?

Scientists use viruses as the delivery vectors to carry on the desired gene and deliver it to the targeted cell.The DNA for the desired gene is inserted into the genetic material of the virus and then the virus itself is inserted into the human cell. .

4.      Can the virus that is used to carry the corrected gene harm my body?

The viruses that are used to carry the desired genes are engineered so they cannot reproduce inside our body, which minimize any bad effects.

5.      Is there any successful cases of gene therapy?  

There are many successful cases in gene therapy including Heredity blindness, Hemophilia, Fat Metabolism disorder, Cancer, Parkinson’s disease.

6.      Is gene therapy available to treat my disorder?

Gene therapy is still in clinical trials, the FDA has not approved to perform gene therapy in humans yet.

Resources: 

http://learn.genetics.utah.edu/content/genetherapy/gtsuccess/

http://ghr.nlm.nih.gov/handbook/therapy

http://www.geogene.com/gene-therapy.html

http://learn.genetics.utah.edu/content/genetherapy/gtchallenges/

http://discovermagazine.com/2009/sep/02-second-coming-of-gene-therapy

https://www.ndsu.edu/pubweb/~mcclean/plsc431/students/bergeson.htm

http://blog.supercoder.com/icd-10-2/cms-offers-assistance-in-transitioning-to-icd-10/