Difference between revisions of "Gene Therapy versus Human Genome Editing (Biotechnology and Society 2016)"

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Revision as of 13:26, 30 October 2016


Glybera (Alipogene tiparvovec) gene therapy

Download Pre-Discussion Questions (MS Word)

Download Policy Czar Template (MS Word)

Everyone: SCID

Required Reading/Viewing

Additional Resources


Especially, check out the "Tools of the Trade" and "Space Doctor" links.

Familial Lipoprotein Lipase Deficiency (LPLD) Czar

Required Reading/Viewing

  • Bryant, L.M., Christopher, D.M., Giles, A.R., Hinderer, C., Rodriguez, J.L., Smith, J.B., Traxler, E.A., Tycko, J., Wojno, A.P., and Wilson, J.M. (2013). Lessons learned from the clinical development and market authorization of Glybera. Hum. Gene Ther. Clin. Dev. 24:55–64.
    Chronicles the development of the first commercially available gene therapy treatment (available in the U.S. in fall 2014).
  • Glybera Glybera - commercial gene therapy product available in Europe.
Error fetching PMID 21443272:
  1. Error fetching PMID 21443272: [Griesenbach2011]
    Review of developments in gene therapy for cystic fibrosis over the last ~20 years.

SCID (Severe Combined Immunodeficiency Disease) Czar

Required Reading/Viewing

  • Hacein-Bey-Abina, S., Garrigue, A., Wang, G.P., Soulier, J., Lim, A., Morillon, E., Clappier, E., Caccavelli, L., Delabesse, E., Beldjord, K., et al. (2008). Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J. Clin. Invest. 118:3132–3142.