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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
2003 2
2005 1
2007 1
2008 3
2009 4
2010 1
2011 2
2013 3
2014 1
2015 7
2016 5
2017 10
2018 3
2019 6
2020 3
2021 2
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47 results
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Page 1
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.
Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, McCague S, Cross D, Marshall KA, Walshire J, Kehoe TL, Reichert H, Davis M, Raffini L, George LA, Hudson FP, Dingfield L, Zhu X, Haller JA, Sohn EH, Mahajan VB, Pfeifer W, Weckmann M, Johnson C, Gewaily D, Drack A, Stone E, Wachtel K, Simonelli F, Leroy BP, Wright JF, High KA, Maguire AM. Russell S, et al. Lancet. 2017 Aug 26;390(10097):849-860. doi: 10.1016/S0140-6736(17)31868-8. Epub 2017 Jul 14. Lancet. 2017. PMID: 28712537 Free PMC article. Clinical Trial.
Cellular therapy: Immune-related complications.
Oved JH, Barrett DM, Teachey DT. Oved JH, et al. Immunol Rev. 2019 Jul;290(1):114-126. doi: 10.1111/imr.12768. Immunol Rev. 2019. PMID: 31355491 Free PMC article. Review.
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hui D, Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA. George LA, et al. N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538. N Engl J Med. 2017. PMID: 29211678 Free PMC article. Clinical Trial.
Hemophilia gene therapy comes of age.
George LA. George LA. Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):587-594. doi: 10.1182/asheducation-2017.1.587. Hematology Am Soc Hematol Educ Program. 2017. PMID: 29222308 Free PMC article. Review.
Hemophilia gene therapy comes of age.
George LA. George LA. Blood Adv. 2017 Dec 8;1(26):2591-2599. doi: 10.1182/bloodadvances.2017009878. eCollection 2017 Dec 12. Blood Adv. 2017. PMID: 29296912 Free PMC article. Review.
47 results
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