Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
Author:
Publisher
Springer Science and Business Media LLC
Subject
Genetics (clinical),Genetics,Molecular Biology
Link
http://www.nature.com/articles/nrg2988.pdf
Reference176 articles.
1. McCandless, S. E., Brunger, J. W. & Cassidy, S. B. The burden of genetic disease on inpatient care in a children's hospital. Am. J. Hum. Genet. 74, 121–127 (2004).
2. Emery, A. E. H. & Rimoin, D. L. Principles and Practice of Medical Genetics 2nd edn (Churchill Livingstone, New York, 1990).
3. Aiuti, A. et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N. Engl. J. Med. 360, 447–458 (2009).
4. Bainbridge, J. W. et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N. Engl. J. Med. 358, 2231–2239 (2008). References 3 and 4 (together with references 7 and 17) are the first reports of efficacy in studies of AAV gene transfer in subjects with RPE65 deficiency.
5. Cartier, N. et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 326, 818–823 (2009).
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