Gene Therapy (Biotechnology and Society)

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

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

Additional Resources
// Review of developments in gene therapy for cystic fibrosis over the last ~20 years.
 * 1) Griesenbach2011 pmid=21443272
 * 1) Glybera Glybera - commercial gene therapy product available in Europe.