It's time for gene therapy to get disruptive!

Human Gene Therapy

Volumn 23, Issue 1, 2012, Pages 1-3

  Wilson, James M ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

PARVOVIRUS VECTOR; PEGADEMASE;

ADRENOLEUKODYSTROPHY; ALLOGENIC BONE MARROW TRANSPLANTATION; COST BENEFIT ANALYSIS; GENE THERAPY; HEMOPHILIA B; HUMAN; NOTE; REIMBURSEMENT; VIRAL GENE THERAPY; X LINKED SEVERE COMBINED IMMUNODEFICIENCY;

DRUG INDUSTRY; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC DISEASES, INBORN; HUMANS;

EID: 84862908972     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2011.2530     Document Type: Note

References (16)

1. Aarkstore. (2010). Hemophilia-Pipeline Assessment and Market Forecasts to 2017; 31 Dec. 2010. www.aarkstore.com/reports/Hemophilia-Pipeline-Assessment- and-Market-Forecasts-to-2017-92008.html (accessed on 1/4/12).
2. Bainbridge, J.W., Smith, A.J., Barker, S.S., et al. (2008). Effect of gene therapy on visual function in Leber's congenital amau-rosis. N. Engl. J. Med. 358, 2231-2239. (Pubitemid 351724452)
3. Bower, J.L., and Christensen, C.M. (1995). Disruptive technologies-catching the wave. Harvard Business Rev. 73, 43-53.
4. Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C.C., et al. (2009). Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 326, 818-823.
5. Christensen, C.M. (1997). The Innovator's Dilemma. (Harvard Business School Press, Boston, MA).
6. Christensen, C.M., and Raynor, M.E. (2003). The Innovator's Solution. (Harvard Business School Press, Boston, MA).
7. Cideciyan, A.V., Hauswirth, W.W., Aleman, T.S., et al. (2009). Human RPE65 gene therapy for Leber congenital amaurosis: Persistence of early visual improvements and safety at 1 year. Hum. Gene Ther. 20, 999-1004.
8. Enzon Pharmaceuticals. (2009). 2009 Form 10-K Annual Report. (United States Securities and Exchange Commission, Washington, D.C.), p. 41.
9. Friedrich, O. (1983). The computer moves in. Time, January 3, 1983, p. 21.
10. Gartner. (2006). Gartner Says Worldwide PC Shipment Growth to Slow in 2006 STAMFORD, Conn., March 9, 2006. www .gartner.com/press-releases/asset- 146531-11.html (accessed 01/05/12).
11. Gaspar, H.B., Cooray, S., Gilmour, K.C., et al. (2011). Hemato-poietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction. Sci. Transl. Med. 3, 97ra80.
12. Hacein-Bey-Abina, S., Hauer, J., Lim, A., et al. (2010). Efficacy of gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 363, 355-364.
13. Maguire, A.M., Simonelli, F., Pierce, E.A., et al. (2008). Safety and efficacy of gene transfer for Leber's congenital amaurosis. N. Engl. J. Med. 358, 2240-2248. (Pubitemid 351724453)
14. Manco-Johnson, et al. (2007). Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N. Engl. J. Med. 357, 535-544. (Pubitemid 47236278)
15. Nathwani, A.C., Tuddenham, E.G., Rangarajan, S., et al. (2011). Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N. Engl. J. Med. 365, 2357-2365.
16. Woodcock, J. (2011). PDUFA V: Medical Innovations, Jobs, and Patients. Statement of Janet Woodcock, M.D., Director, Center for Drug Evaluation and Research, Food and Drug Administration, Department of Health and Human Services before the Subcommittee on Health, Committee on Energy and Commerce, U.S. House of Representatives, July 7, 2011. Available at www.fda.gov/NewsEvents/Testimony/ ucm261396 .htm