Gene Therapy

Anna Crawford Stephanie Dasai Ben Biggs Ryan Creeekmur Rita Arnold

What is Gene Therapy?

Gene therapy is a new and experimental technique used to correct defective genes that cause diseases and genetic disorders.

This therapy can be used to prevent or cure various genetic disorders, however, it is still risky and it is only tested on diseases that have no other cure. http://youtu.be/CXPRu1WhxSs

Cystic fibrosis Sickle cell anemia Spinal muscular atrophy

Techniques
Insert

a normal gene to replace the dysfunctional gene Use homologous recombination to switch the abnormal gene for a normal gene Repair abnormal gene through reverse mutation to return the gene to its normal state The regulation of the gene can be altered

Why isnt it more common?

Therapy is short-lived

Immune Response

The nature of gene therapy would require a patient to undergo multiple rounds. Multiple rounds illicit an immune response from the body which attacks a foreign body that is introduced

Viruses

Multi-gene Disorders

Viruses are often used in gene therapy but can be harmful to the patient
Patients with more than one genetic disease can be very difficult to treat with gene therapy.

How it Works
An

non-inflicted gene is introduced into a system to replace genes inflicted with disease. New genes are introduced through vectors that are usually genetically altered viruses.

Vector Viruses

Retroviruses

Adenoviruses

Create double stranded DNA copies of RNA genomes

Adeno-associated viruses

Have double stranded DNA genomes that cause infections in the eyes, intestines, and lungs
Have single stranded DNA and insert it on the 19th chromosome Have double stranded DNA and infect neurons.

Herpes simplex virus

Other methods

The non-inflicted genes are introduced directly into the cell. Creation of an artificial lipid sphere, a liposome, that carries non-inflicted DNA through the cell membrane. Non-inflicted DNA is linked chemically to a molecule that binds to certain cell receptors; the DNA is then surrounded by the membrane and enters the cell. Introduction of an artificial 47th chromosome that would carry therapeutic DNA and not interfere with other genes.

Vector Virus Gene Therapy

Gene Insertion Gene Therapy

In Todays current study

FDA

has not yet approved any human gene therapy product for sale. It also has not been proven successful in clinical trails. Little progress has been made since first clinical trial in 1990. A few deaths due to gene therapy has set back most of the research.

Graph link

Recent developments on gene therapy research

March

2009- London April 2008- UK February 2005 - US

Positive potential
Using gene therapy and the technology associated with gene therapy, scientists are working towards the prevention of and cures for: Muscular Dystrophy Sickle Cell Anemia Hemophilia

Negative Potential
Even with all the positive potential gene therapy has, there are still some concerns. Could permanently alter the human gene pool. Is it ethical? Could create new, possibly worse, conditions.

How has Gene Therapy Changed the World?

Although

gene therapy is still in an experimental state, it has the potential to change lives. There are many problems associated with gene therapy both ethical and medical, but once the techniques of gene therapy are perfected, it has the ability to cure genetic diseases that have disabled millions.

Sources

U.S. Department of Energy Genome Program's Biological and Environmental Research Information System (BERIS). (August 24, 2011). Gene Therapy. In Human Genome Project Information. Retrieved 11/21/2011, from http://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.sh tml. Pharmaceutical Research & Development Pipeline Intelligence. (April, 2009). Therapy Analysis - Gene therapy - Still the Next Big Thing?. In Pharma Projects. Retrieved 11/21/2011, from http://www.pharmaprojects.com/therapy_analysis/genether_current_0409.html. Human Genome Project Information Genetics Home Reference Hogarth, Eric. "Aspects of Gene Therapy (Pros & Cons)." Web. 21 Nov. 2011. <http://www.ndsu.edu/pubweb/~mcclean/plsc431/students/eric.htm>. http://ghr.nlm.nih.gov/handbook/therapy/genetherapy http://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.sh tml#factors