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Items: 1 to 20 of 86

1.

Production and Purification of Baculovirus for Gene Therapy Application.

Nasimuzzaman M, van der Loo JCM, Malik P.

J Vis Exp. 2018 Apr 9;(134). doi: 10.3791/57019.

2.

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.

N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538.

3.

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.

Hum Gene Ther Clin Dev. 2017 Dec;28(4):178-186. doi: 10.1089/humc.2017.170. Epub 2017 Nov 27.

4.

Foamy Virus Vector Carries a Strong Insulator in Its Long Terminal Repeat Which Reduces Its Genotoxic Potential.

Goodman MA, Arumugam P, Pillis DM, Loberg A, Nasimuzzaman M, Lynn D, van der Loo JCM, Dexheimer PJ, Keddache M, Bauer TR Jr, Hickstein DD, Russell DW, Malik P.

J Virol. 2017 Dec 14;92(1). pii: e01639-17. doi: 10.1128/JVI.01639-17. Print 2018 Jan 1.

5.

Purification of baculovirus vectors using heparin affinity chromatography.

Nasimuzzaman M, Lynn D, van der Loo JC, Malik P.

Mol Ther Methods Clin Dev. 2016 Dec 7;3:16071. eCollection 2016.

6.

Production and purification of high-titer foamy virus vector for the treatment of leukocyte adhesion deficiency.

Nasimuzzaman M, Lynn D, Ernst R, Beuerlein M, Smith RH, Shrestha A, Cross S, Link K, Lutzko C, Nordling D, Russell DW, Larochelle A, Malik P, Van der Loo JC.

Mol Ther Methods Clin Dev. 2016 Mar 16;3:16004. eCollection 2016.

7.

Progress and challenges in viral vector manufacturing.

van der Loo JC, Wright JF.

Hum Mol Genet. 2016 Apr 15;25(R1):R42-52. doi: 10.1093/hmg/ddv451. Epub 2015 Oct 30. Review.

8.

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.

N Engl J Med. 2014 Oct 9;371(15):1407-17. doi: 10.1056/NEJMoa1404588.

9.

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.

Gene Ther. 2012 Aug;19(8):872-6. doi: 10.1038/gt.2012.37. Epub 2012 May 3.

10.

Scale-up and manufacturing of clinical-grade self-inactivating γ-retroviral vectors by transient transfection.

van der Loo JC, Swaney WP, Grassman E, Terwilliger A, Higashimoto T, Schambach A, Baum C, Thrasher AJ, Williams DA, Nordling DL, Reeves L, Malik P.

Gene Ther. 2012 Mar;19(3):246-54. doi: 10.1038/gt.2011.102. Epub 2011 Jul 14.

11.

Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease.

Nicolino M, Byrne B, Wraith JE, Leslie N, Mandel H, Freyer DR, Arnold GL, Pivnick EK, Ottinger CJ, Robinson PH, Loo JC, Smitka M, Jardine P, Tatò L, Chabrol B, McCandless S, Kimura S, Mehta L, Bali D, Skrinar A, Morgan C, Rangachari L, Corzo D, Kishnani PS.

Genet Med. 2009 Mar;11(3):210-9. doi: 10.1097/GIM.0b013e31819d0996.

PMID:
19287243
12.

Design and production of retro- and lentiviral vectors for gene expression in hematopoietic cells.

Schambach A, Swaney WP, van der Loo JC.

Methods Mol Biol. 2009;506:191-205. doi: 10.1007/978-1-59745-409-4_14.

PMID:
19110628
13.

Familial pulmonary alveolar proteinosis caused by mutations in CSF2RA.

Suzuki T, Sakagami T, Rubin BK, Nogee LM, Wood RE, Zimmerman SL, Smolarek T, Dishop MK, Wert SE, Whitsett JA, Grabowski G, Carey BC, Stevens C, van der Loo JC, Trapnell BC.

J Exp Med. 2008 Nov 24;205(12):2703-10. doi: 10.1084/jem.20080990. Epub 2008 Oct 27.

14.

PU.1 redirects adenovirus to lysosomes in alveolar macrophages, uncoupling internalization from infection.

Carey B, Staudt MK, Bonaminio D, van der Loo JC, Trapnell BC.

J Immunol. 2007 Feb 15;178(4):2440-7.

15.

Search for genetic variants associated with cutaneous malignant melanoma in the Ashkenazi Jewish population.

Loo JC, Paterson AD, Hao A, Shennan M, Peretz H, Sidi Y, Hogg D, Yakobson E.

J Med Genet. 2005 May;42(5):e30. No abstract available.

16.

Germline splicing mutations of CDKN2A predispose to melanoma.

Loo JC, Liu L, Hao A, Gao L, Agatep R, Shennan M, Summers A, Goldstein AM, Tucker MA, Deters C, Fusaro R, Blazer K, Weitzel J, Lassam N, Lynch H, Hogg D.

Oncogene. 2003 Sep 25;22(41):6387-94.

PMID:
14508519
17.

Homing defect of cultured human hematopoietic cells in the NOD/SCID mouse is mediated by Fas/CD95.

Liu B, Buckley SM, Lewis ID, Goldman AI, Wagner JE, van der Loo JC.

Exp Hematol. 2003 Sep;31(9):824-32.

PMID:
12962729
18.

Ambiguity and mistakes in the pediatric cardiology vernacular.

Sklansky MS, Loo JC, Rothman A.

Pediatr Cardiol. 2002 Nov-Dec;23(6):577-9.

PMID:
12530487
20.

Historical perspectives on thrombolytic therapy.

van de Loo JC.

Thromb Haemost. 1999 Aug;82(2):971-3. No abstract available.

PMID:
10605811

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