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Progress and challenges in viral vector manufacturing.
van der Loo JC, Wright JF. van der Loo JC, et al. 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.
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: van der loo jcm. 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.
The Gene Therapy Resource Program: A Decade of Dedication to Translational Research by the National Heart, Lung, and Blood Institute.
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. Flotte TR, et al. Among authors: van der loo jcm. Hum Gene Ther Clin Dev. 2017 Dec;28(4):178-186. doi: 10.1089/humc.2017.170. Epub 2017 Nov 27. Hum Gene Ther Clin Dev. 2017. PMID: 29130351 Free PMC article.
Preclinical Evaluation of Foamy Virus Vector-Mediated Gene Addition in Human Hematopoietic Stem/Progenitor Cells for Correction of Leukocyte Adhesion Deficiency Type 1.
Smith RH, Bloomer H, Fink D, Keyvanfar K, Nasimuzzaman M, Sancheznieto F, Dutta R, Guenther Bui K, Alvarado LJ, Bauer TR Jr, Hickstein DD, Russell DW, Malik P, van der Loo JCM, Highfill SL, Kuhns DB, Pirooznia M, Larochelle A. Smith RH, et al. Among authors: van der loo jcm. Hum Gene Ther. 2022 Dec;33(23-24):1293-1304. doi: 10.1089/hum.2022.065. Epub 2022 Nov 1. Hum Gene Ther. 2022. PMID: 36094106 Free PMC article.
Selecting a Cell Engineering Methodology During Cell Therapy Product Development.
Timmins LM, Burr AM, Carroll K, Keefe R, Teryek M, Cantolupo LJ, van der Loo JCM, Heathman TRJ, Gormley A, Smith D, Parekkadan B. Timmins LM, et al. Among authors: van der loo jcm. Cell Transplant. 2021 Jan-Dec;30:9636897211003022. doi: 10.1177/09636897211003022. Cell Transplant. 2021. PMID: 34013781 Free PMC article. Review.
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.
Hacein-Bey-Abina S, Pai SY, Gaspar HB, Armant M, Berry CC, Blanche S, Bleesing J, Blondeau J, de Boer H, Buckland KF, Caccavelli L, Cros G, De Oliveira S, Fernández KS, Guo D, Harris CE, Hopkins G, Lehmann LE, Lim A, London WB, van der Loo JC, Malani N, Male F, Malik P, Marinovic MA, McNicol AM, Moshous D, Neven B, Oleastro M, Picard C, Ritz J, Rivat C, Schambach A, Shaw KL, Sherman EA, Silberstein LE, Six E, Touzot F, Tsytsykova A, Xu-Bayford J, Baum C, Bushman FD, Fischer A, Kohn DB, Filipovich AH, Notarangelo LD, Cavazzana M, Williams DA, Thrasher AJ. Hacein-Bey-Abina S, et al. Among authors: van der loo jc. N Engl J Med. 2014 Oct 9;371(15):1407-17. doi: 10.1056/NEJMoa1404588. N Engl J Med. 2014. PMID: 25295500 Free PMC article. Clinical Trial.
Critical variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency.
van der Loo JC, Swaney WP, Grassman E, Terwilliger A, Higashimoto T, Schambach A, Hacein-Bey-Abina S, Nordling DL, Cavazzana-Calvo M, Thrasher AJ, Williams DA, Reeves L, Malik P. van der Loo JC, et al. Gene Ther. 2012 Aug;19(8):872-6. doi: 10.1038/gt.2012.37. Epub 2012 May 3. Gene Ther. 2012. PMID: 22551777 Free PMC article.
35 results