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

Year Number of Results
2002 1
2003 4
2004 1
2005 1
2006 3
2007 3
2008 3
2009 6
2010 6
2011 7
2012 6
2013 6
2014 5
2015 3
2016 4
2017 2
2018 1
2019 3
2020 5
2021 5
2022 2
2023 4
2024 1

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73 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. Among authors: wright jf. 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.
Chimeric antigen receptor-modified T cells for acute lymphoid leukemia.
Grupp SA, Kalos M, Barrett D, Aplenc R, Porter DL, Rheingold SR, Teachey DT, Chew A, Hauck B, Wright JF, Milone MC, Levine BL, June CH. Grupp SA, et al. Among authors: wright jf. N Engl J Med. 2013 Apr 18;368(16):1509-1518. doi: 10.1056/NEJMoa1215134. Epub 2013 Mar 25. N Engl J Med. 2013. PMID: 23527958 Free PMC article.
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. Among authors: wright jf. 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.
Safety and efficacy of gene transfer for Leber's congenital amaurosis.
Maguire AM, Simonelli F, Pierce EA, Pugh EN Jr, Mingozzi F, Bennicelli J, Banfi S, Marshall KA, Testa F, Surace EM, Rossi S, Lyubarsky A, Arruda VR, Konkle B, Stone E, Sun J, Jacobs J, Dell'Osso L, Hertle R, Ma JX, Redmond TM, Zhu X, Hauck B, Zelenaia O, Shindler KS, Maguire MG, Wright JF, Volpe NJ, McDonnell JW, Auricchio A, High KA, Bennett J. Maguire AM, et al. Among authors: wright jf. N Engl J Med. 2008 May 22;358(21):2240-8. doi: 10.1056/NEJMoa0802315. Epub 2008 Apr 27. N Engl J Med. 2008. PMID: 18441370 Free PMC article. Clinical Trial.
Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease.
Lattanzi A, Camarena J, Lahiri P, Segal H, Srifa W, Vakulskas CA, Frock RL, Kenrick J, Lee C, Talbott N, Skowronski J, Cromer MK, Charlesworth CT, Bak RO, Mantri S, Bao G, DiGiusto D, Tisdale J, Wright JF, Bhatia N, Roncarolo MG, Dever DP, Porteus MH. Lattanzi A, et al. Among authors: wright jf. Sci Transl Med. 2021 Jun 16;13(598):eabf2444. doi: 10.1126/scitranslmed.abf2444. Sci Transl Med. 2021. PMID: 34135108 Free PMC article.
Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials.
Maguire AM, Russell S, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, Marshall KA, McCague S, Reichert H, Davis M, Simonelli F, Leroy BP, Wright JF, High KA, Bennett J. Maguire AM, et al. Among authors: wright jf. Ophthalmology. 2019 Sep;126(9):1273-1285. doi: 10.1016/j.ophtha.2019.06.017. Epub 2019 Jun 22. Ophthalmology. 2019. PMID: 31443789 Free article. Clinical Trial.
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.
Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Rustagi PK, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA, Kay MA. Manno CS, et al. Among authors: wright jf. Nat Med. 2006 Mar;12(3):342-7. doi: 10.1038/nm1358. Epub 2006 Feb 12. Nat Med. 2006. PMID: 16474400 Clinical Trial.
Progress and challenges in viral vector manufacturing.
van der Loo JC, Wright JF. van der Loo JC, et al. Among authors: wright jf. Hum Mol Genet. 2016 Apr 15;25(R1):R42-52. doi: 10.1093/hmg/ddv451. Epub 2015 Oct 30. Hum Mol Genet. 2016. PMID: 26519140 Free PMC article. Review.
The intersection of vector biology, gene therapy, and hemophilia.
Lisowski L, Staber JM, Wright JF, Valentino LA. Lisowski L, et al. Among authors: wright jf. Res Pract Thromb Haemost. 2021 Sep 1;5(6):e12586. doi: 10.1002/rth2.12586. eCollection 2021 Aug. Res Pract Thromb Haemost. 2021. PMID: 34485808 Free PMC article. Review.
73 results