Gene Therapy (Biotechnology and Society)

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Download Pre-Discussion Questions (PDF)

Glybera (Alipogene tiparvovec) gene therapy

Required Reading/Viewing

We will discuss gene therapy for two diseases: SCID (Severe Combined Immunodeficiency Disease) and Familial Lipoprotein Lipase Deficiency (LPLD).

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Error fetching PMID 23808604:
  1. Zimmer. Gene Therapy emerges from disgrace to be the next big thing, again. Wired Science Blogs. August 13, 2013. [Carl]
    Read article at
  2. Gene Therapy: Molecular Bandage? Educational website at the University of Utah. [Bandage2013]
    Especially, check out the "Tools of the Trade" and "Space Doctor" links.
  3. Error fetching PMID 18688285: [Hacein2008]
    Study characterizing the genomes of SCID patients who developed Leukemia after successful gene therapy. Feel free to skim for key details.
  4. Error fetching PMID 23808604: [Bryant2013]
    Chronicles the development of the first commercially available gene therapy treatment (available in the U.S. in fall 2014).
  5. ISBN:8847016428 [Giacca2010]
    Read Chapter 5: Ethical and Social Problems of Gene Therapy
All Medline abstracts: PubMed | HubMed

Additional Resources

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  1. Error fetching PMID 21443272: [Griesenbach2011]
    Review of developments in gene therapy for cystic fibrosis over the last ~20 years.
  2. Glybera - commercial gene therapy product available in Europe. [Glybera]