charlie mclaughlin. what are genetic disorders? genetic disorders are illnesses stemming from...
TRANSCRIPT
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.