Clinical Vector Core Publications



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Below is a list of publications by researchers who used resources in the Clinical Vector Core Lab. To have your reference possibly featured on this page, send your paper to

Kuzmin DA, Shutova MV, Johnston NR, Smith OP, Fedorin VV, Kukushkin YS, van der Loo JCM and Johnstone EC. The clinical landscape for AAV gene therapies. Nat. Rev. Drug Discov. 2021 Jan; doi: 10.1038/d41573-021-00017-7
Flotte TR, Daniels E, Benson J, Bevett-Rose JM, Cornetta K, Diggins M, Johnston J, Sepelak S, van der Loo JCM, Wilson JM, McDonald CL. The Gene Therapy Resource Program: A Decade of Dedication to Translational Research by the National Heart, Lung, and Blood Institute. Hum Gene Ther Clin Dev. 2017 Dec; 28(4):178-186. PMID: 29130351
van der Loo JCM, Wright JF. Progress and challenges in viral vector manufacturing. Hum Mol Genet. 2015 Oct; PMID: 26519140 PMCID: PMC4802372 DOI: 10.1093/hmg/ddv451
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. Chimeric antigen receptor-modified T cells for acute lymphoid leukemia. N Engl J Med. 2013 Apr; 368(16):1509-1518. doi: 10.1056/NEJMoa1215134. Epub 2013 Mar 25. Erratum in: N Engl J Med. 2016 Mar 10;374(10):998. PubMed PMID: 23527958; PubMed Central PMCID: PMC4058440
Wright JF, and Zelenaia O. Vector characterization methods for quality control testing of recombinant adeno-associated viruses. Methods Mol Biol. 2011 Jan; 737(247-78. PMID: 21590401
Hauck B, Murphy SL, Smith PH, Qu G, Liu X, Zelenaia O, Mingozzi F, Sommer JM, High KA, and Wright JF. Undetectable transcription of cap in a clinical AAV vector: implications for preformed capsid in immune responses. Mol Ther. 2008 Oct; 17(1):144-52. PMID: 18941440
Maguire AM, Simonelli F, Pierce EA, Pugh EN, Jr., Mingozzi F, Bennicelli J, Banfi S, Marshall KA, Testa F, Surace EM, et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med. 2008 Apr; 358(21): 2240-8. PMID: 18441370
Bennicelli J, Wright JF, Komaromy A, Jacobs JB, Hauck B, Zelenaia O, Mingozzi F, Hui D, Chung D, Rex TS, et al. Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer. Mol Ther. 2008 Jan; 16(3):458-65. PMID: 18209734