Gene Therapy for Genetic Disorders

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Gene Therapy for Genetic Disorders

• Charlie McLaughlin

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What are Genetic Disorders?

• Genetic disorders are illnesses stemming from
  errors in a persons 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

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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

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What is Gene Therapy?

• Gene therapy is the process of taking DNA coded
  for specific genes and adding it to a persons
  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

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In Vivo Vs. Ex Vivo
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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

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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

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Non Viral Vectors (cont.)
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Limitations

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

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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

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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 lthttp//en.wikipedia.org/w
  iki/Gene_therapygt.
• Wikipedia Genetic Disorders lthttp//en.wikipedia.
  org/wiki/Gene_disordersgt.
• 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 6577, February 2013.