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New England Journal of Medicine, ISSN 0028-4793, 05/2015, Volume 372, Issue 20, pp. 1887 - 1897
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The New England Journal of Medicine, ISSN 0028-4793, 05/2008, Volume 358, Issue 21, pp. 2240 - 2248
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Nature Genetics, ISSN 1061-4036, 09/2012, Volume 44, Issue 9, pp. 1040 - 1045
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Proceedings of the National Academy of Sciences, ISSN 0027-8424, 02/2013, Volume 110, Issue 6, pp. E517 - E525
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The New England Journal of Medicine, ISSN 0028-4793, 05/2008, Volume 358, Issue 21, pp. 2231 - 2239
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Gene Therapy, ISSN 0969-7128, 2014, Volume 21, Issue 7, pp. 662 - 672
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Lancet, The, ISSN 0140-6736, 2009, Volume 374, Issue 9701, pp. 1597 - 1605
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