Clinical Vector Core Publications


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

Heizel M, Suzuki T, Hashtchin AR, Arumugam P, Carey B, Schwabbauer M, Kuhn A, Meyer J, Schambach A, van der Loo J, Moritz T, Trapnell BC, Lachmann. Function and Safety of Lentivirus-Mediated Gene Transfer for CSF2RA-Deficiency. N Hum Gene Ther Methods. 2017 Dec 28; 28(6):318-329. PMID: 28854814
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 01; 28(4):178-186. PMID: 29130351
Nasimuzzaman M, Lynn D, Ernst R, Beuerlein M, Smith RH, Cross S, Link K, Lutzko C, Nordling D, Russell DW, Larochelle A, Malik P, van der Loo. Production and purification of high-titer foamy virus vector for the treatment of leukocyte adhesion deficiency. JCM Mol Ther Methods Clin Dev. 2016 Oct 03; 3:16004. PMID: 27722179
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 18; 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 12; 737(247-78. PMID: 21590401
Maguire AM, High KA, Auricchio A, Wright JF, Pierce EA, Testa F, Mingozzi F, Bennicelli JL, Ying GS, Rossi S, et al. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet. 2009 Oct 23; 2009;374(9701):1597-605. PMID: 19854499
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 22; 16(3):458-65. PMID: 18209734
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 21; 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 27; 358(21): 2240-8. PMID: 18441370
Qu G, Bahr-Davidson J, Prado J, Tai A, Cataniag F, McDonnell J, Zhou J, Hauck B, Luna J, Sommer JM, et al. Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography. J Virol Methods. 2006 Dec 28; 140(1-2):183-92. PMID: 17196264