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

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(Required Reading/Viewing)
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== Everyone: SCID ==
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== Everyone: Overview of gene therapy, germline modification, and genome synthesis ==
  
 
== Required Reading/Viewing ==
 
== Required Reading/Viewing ==
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* [http://link.springer.com/chapter/10.1007/978-88-470-1643-9_5 Read Chapter 5: Ethical and Social Problems of Gene Therapy]<br>Access from on-campus to read.
 
* [http://link.springer.com/chapter/10.1007/978-88-470-1643-9_5 Read Chapter 5: Ethical and Social Problems of Gene Therapy]<br>Access from on-campus to read.
  
== Familial Lipoprotein Lipase Deficiency (LPLD) Czar ==
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== CRISPR Germline Modification Czar ==
  
==== Required Reading/Viewing ====
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* [http://gizmodo.com/everything-you-need-to-know-about-crispr-the-new-tool-1702114381 Everything You Need to Know About CRISPR, the New Tool that Edits DNA] Sarah Zhang ''Gizmodo'' May 6, 2015.
 
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* Lanphier, E., Urnov, F., Haecker, S.E., Werner, M., and Smolenski, J. (2015). [http://www.nature.com/news/don-t-edit-the-human-germ-line-1.17111 Don’t edit the human germ line.] ''Nature'' '''519''': 410–411.
 
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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 (available in the U.S. in fall 2014).
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* Glybera [http://www.uniqure.com/products/glybera/ Glybera] - commercial gene therapy product available in Europe.
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<biblio>
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#Griesenbach2011 pmid=21443272
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// Review of developments in gene therapy for cystic fibrosis over the last ~20 years.
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</biblio>
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== SCID (Severe Combined Immunodeficiency Disease) Czar ==
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== Gene Therapy Czar ==
  
 
==== Required Reading/Viewing ====
 
==== 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. <br>
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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).
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* Glybera [http://www.uniqure.com/products/glybera/ Glybera] - commercial gene therapy product.
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* 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 13:38, 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 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.