Cavazzano-Calvo, M. (2010). Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. Nature 467, 318-322. doi:10.1038/nature09328

Cideciyan, A.V. et al (22 January 2013). Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement. Proceedings of the National Academy of Sciences of the United States of America, Earline Online Publication. doi: 10.1073/pnas.1218933110

MacLaren, R.E. et al (16 January 2014). Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial. The Lancet, Early Online Publication. doi:10.1016/S0140-6736(13)62117-0

Nathwani, A.C. (2011). Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. The New England Journal of Medicine, 365(25), 2357-2365.

Nienhuis, A.W. (2013). Development of gene therapy for blood disorders: an update. Blood 122(9), 1556-1564. doi: 10.1182/blood-2013-04-453209

Palfi, S. et al (10 January 2014). Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for parkinson's disease; a dose escalation, open-label, phase 1/2 trial. The Lancet, Early Online Publication. doi: 10.1016/S014006736(13)61939-X

Penn Medicine (7 December 2013). Penn medicine team reports findings from research study of first 59 adult and pediatric leukemia patients who received investigational, personalized cellular therapy CTL019. Retrieved from http://www.uphs.upenn.edu/news/News_Releases/2013/12/ctl019/

Persons, Derek A. (2010). Gene therapy: Targeting beta-thalassaemia. Nature 467, 277-278. doi: 10.1038/467277a

Petrs-Silva, H. & R. Linden (2014). Advances in gene therapy technologies to treat retinitis pigmentosa. Clinical Opthalmology 2014(8), 127-136. doi: 10.2147/OPTH.538041

Read more from the original source:

Gene Therapy Successes - University of Utah