Gene Therapy (Biotechnology and Society)
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Required Reading/Viewing
We will concentrate on gene therapy for two diseases: SCID (Severe Combined Immunodeficiency Disease) and Familial Lipoprotein Lipase Deficiency (LPLD).
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- Zimmer. Gene Therapy Emerged from Disgrace to Be the Next Big Thing, Again. Wired Science Blogs. August 13, 2013.
Read article at wired.com. - Gene Therapy: Molecular Bandage? Educational website at the University of Utah.
Especially, check out the "Tools of the Trade" and "Space Doctor" links. - Error fetching PMID 18688285:
Study characterizing genomes SCID patients who developed Leukemia after successful gene therapy. - Error fetching PMID 23808604:
Chronicle of development of the first commercially available gene therapy treatment, available in the U.S. in fall 2014. - ISBN:8847016428
Additional Resources
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Review of development of gene therapy for cystic fibrosis over the last ~20 years. - Glybera - commercial gene therapy product available in Europe.