Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders
Author:
Publisher
Wiley
Subject
Genetics (clinical),Genetics
Link
http://www.springerlink.com/index/pdf/10.1007/s10545-012-9467-0
Reference38 articles.
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4. Bainbridge JW, Smith AJ, Barker SS et al (2008) Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 358:2231–2239
5. Boztug K, Schmidt M, Schwarzer A et al (2010) Stem-cell gene therapy for the Wiskott-Aldrich syndrome. N Engl J Med 363:1918–1927
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