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

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(Gene Therapy Czar)
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* 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). [https://www.ncbi.nlm.nih.gov/pubmed/23808604 Lessons learned from the clinical development and market authorization of Glybera.] ''Hum. Gene Ther. Clin. Dev.'' '''24''':55–64. <br>Chronicles the development of the first commercially available gene therapy treatment for Familial Lipoprotein Lipase Deficiency (LPLD).
 
* 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). [https://www.ncbi.nlm.nih.gov/pubmed/23808604 Lessons learned from the clinical development and market authorization of Glybera.] ''Hum. Gene Ther. Clin. Dev.'' '''24''':55–64. <br>Chronicles the development of the first commercially available gene therapy treatment for Familial Lipoprotein Lipase Deficiency (LPLD).
* Glybera [http://www.uniqure.com/products/glybera/ Glybera] - commercial gene therapy product.
+
*[http://www.uniqure.com/products/glybera/ Glybera] - commercial gene therapy product.
 
* 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. <br>Scan for the major take-home message.
 
* 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. <br>Scan for the major take-home message.

Revision as of 13:39, 30 October 2016


Glybera (Alipogene tiparvovec) gene therapy

Download Pre-Discussion Questions (MS Word)

Download Policy Czar Template (MS Word)

Everyone: Overview of gene therapy, germline modification, and genome synthesis

Required Reading/Viewing

Additional Resources

CRISPR Germline Modification Czar

Gene Therapy 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 for Familial Lipoprotein Lipase Deficiency (LPLD).
  • Glybera - commercial gene therapy product.
  • 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.
    Scan for the major take-home message.