Assembly PCR synthesis of optimally designed, compact, multi-responsive promoters suited to gene therapy application
Author:
Publisher
Springer Science and Business Media LLC
Subject
Multidisciplinary
Link
http://www.nature.com/articles/srep29388.pdf
Reference55 articles.
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3. Maguire, A. M. et al. Safety and efficacy of gene transfer for Leber’s congenital amaurosis. N Engl J Med 358, 2240–2248 (2008).
4. Maguire, A. M. et al. Age-dependent effects of RPE65 gene therapy for Leber’s congenital amaurosis: a phase 1 dose-escalation trial. Lancet 374, 1597–1605 (2009).
5. Carpentier, A. C. et al. Effect of alipogene tiparvovec (AAV1-LPL(S447X)) on postprandial chylomicron metabolism in lipoprotein lipase-deficient patients. The Journal of clinical endocrinology and metabolism 97, 1635–1644, 10.1210/jc.2011-3002 (2012).
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