Transcript Gene Therapy for Genetic Disorders

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 cells

 Gold particles laced with DNA
 Electroporation: 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 Transfer
 Jet 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.