Charlie McLaughlin

What are Genetic Disorders? Genetic disorders are illnesses

stemming from errors in a person’s genes

Any mistake in a gene can alter how a specific protein is produced

Without proper proteins, the body will not function properly and will take on a chronic and possibly life threatening condition

How is it Caused?

Genetic disorders can be congenital meaning they occur from birth or they may develop over time

Congenital disorders occur when parents who are carriers of the disorder pass on their genes to their children

Progressive disorders that take time to set in may be caused by a mutation in DNA

What is Gene Therapy?

Gene therapy is the process of taking DNA coded for specific genes and adding it to a person’s genome in efforts to treat and alleviate genetic disorders

There are two types of treatment, In Vivo and Ex Vivo

Regardless of the type, each therapy requires a vector to transport DNA into cells

In Vivo Vs. Ex Vivo

Viral Vectors

Viral vectors are viruses that have had recombinant DNA inserted into their genome

They are injected into patients and infect cells allowing the new therapeutic gene to be copied

The addition of the “fixed” gene will begin to treat the genetic disorder

Non-Viral Vectors

Non-viral vectors are somewhat safer alternatives to viral vectors because they come without an infection risk

They involve different methods of delivery into cellsGold particles laced with DNAElectroporation: electrifying cell membranes

to alter permeability Microbubbles and Ultrasounds

Non Viral Vectors (cont.)

Limitations

Still considered in trial phase Viral Vectors can be infectious Patient specific Under developed technonlogy

Future

Higher success rates in patients Transition to Non viral vectors

Hydrodynamic Gene TransferJet Gun

Ability to cure and not just treat disorders

Ability to cure more diseases

References Edwards, Mark T. Gene Therapy: Current Treatment

Options and Likely Near-Term Developments. Student Pulse: International Student Journal. Vol. 6 NO. 9, 2014.

Wikipedia: Gene Therapy <http://en.wikipedia.org/wiki/Gene_therapy>.

Wikipedia: Genetic Disorders <http://en.wikipedia.org/wiki/Gene_disorders>.

Ginn, Samantha L., Alexander, Ian E., Edelstein, Michael L., Abedi, Mohammad, R., Wixon, Jo. Gene therapy clinical trials worldwide to 2012 – an update. The Journal of Gene Medicine. Vol 15, Iss 2, p 65–77, February 2013.