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Gene Delivery to Human Limbal Stem Cells Using Viral Vectors.

Song L, Song Z, Fry NJ, Conatser L, Llanga T, Mei H, Kafri T, Hirsch ML.

Hum Gene Ther. 2019 Sep 25. doi: 10.1089/hum.2019.071. [Epub ahead of print]


Superior lentiviral vectors designed for BSL-0 environment abolish vector mobilization.

Hu P, Bi Y, Ma H, Suwanmanee T, Zeithaml B, Fry NJ, Kohn DB, Kafri T.

Gene Ther. 2018 Oct;25(7):454-472. doi: 10.1038/s41434-018-0039-2. Epub 2018 Sep 6.


Correction to: Gene therapy knockdown of VEGFR2 in retinal endothelial cells to treat retinopathy.

Simmons AB, Bretz CA, Wang H, Kunz E, Hajj K, Kennedy C, Yang Z, Suwanmanee T, Kafri T, Hartnett ME.

Angiogenesis. 2018 Nov;21(4):765. doi: 10.1007/s10456-018-9626-5.


Gene therapy knockdown of VEGFR2 in retinal endothelial cells to treat retinopathy.

Simmons AB, Bretz CA, Wang H, Kunz E, Hajj K, Kennedy C, Yang Z, Suwanmanee T, Kafri T, Hartnett ME.

Angiogenesis. 2018 Nov;21(4):751-764. doi: 10.1007/s10456-018-9618-5. Epub 2018 May 5. Erratum in: Angiogenesis. 2018 Jun 25;:.


Targeted Knockdown of Overexpressed VEGFA or VEGF164 in Müller cells maintains retinal function by triggering different signaling mechanisms.

Becker S, Wang H, Simmons AB, Suwanmanee T, Stoddard GJ, Kafri T, Hartnett ME.

Sci Rep. 2018 Jan 31;8(1):2003. doi: 10.1038/s41598-018-20278-4.


Toward Personalized Gene Therapy: Characterizing the Host Genetic Control of Lentiviral-Vector-Mediated Hepatic Gene Delivery.

Suwanmanee T, Ferris MT, Hu P, Gui T, Montgomery SA, Pardo-Manuel de Villena F, Kafri T.

Mol Ther Methods Clin Dev. 2017 Apr 5;5:83-92. doi: 10.1016/j.omtm.2017.03.009. eCollection 2017 Jun 16.


Hematopoietic Stem cell transplantation and lentiviral vector-based gene therapy for Krabbe's disease: Present convictions and future prospects.

Hu P, Li Y, Nikolaishvili-Feinberg N, Scesa G, Bi Y, Pan D, Moore D, Bongarzone ER, Sands MS, Miller R, Kafri T.

J Neurosci Res. 2016 Nov;94(11):1152-68. doi: 10.1002/jnr.23847.


Insights into the Pathogenesis and Treatment of Krabbe Disease.

Bongarzone ER, Escolar ML, Gray SJ, Kafri T, Vite CH, Sands MS.

Pediatr Endocrinol Rev. 2016 Jun;13 Suppl 1:689-96. Review.


Post-translational Down-regulation of Melanoma Antigen-A11 (MAGE-A11) by Human p14-ARF Tumor Suppressor.

Minges JT, Grossman G, Zhang P, Kafri T, Wilson EM.

J Biol Chem. 2015 Oct 9;290(41):25174-87. doi: 10.1074/jbc.M115.663641. Epub 2015 Sep 1.


Generation of a stable packaging cell line producing high-titer PPT-deleted integration-deficient lentiviral vectors.

Hu P, Li Y, Sands MS, McCown T, Kafri T.

Mol Ther Methods Clin Dev. 2015 Jul 22;2:15025. doi: 10.1038/mtm.2015.25. eCollection 2015.


Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial.

Monahan PE, Sun J, Gui T, Hu G, Hannah WB, Wichlan DG, Wu Z, Grieger JC, Li C, Suwanmanee T, Stafford DW, Booth CJ, Samulski JJ, Kafri T, McPhee SW, Samulski RJ.

Hum Gene Ther. 2015 Feb;26(2):69-81. doi: 10.1089/hum.2014.106. Epub 2015 Jan 21.


Functional analysis of the putative integrin recognition motif on adeno-associated virus 9.

Shen S, Berry GE, Castellanos Rivera RM, Cheung RY, Troupes AN, Brown SM, Kafri T, Asokan A.

J Biol Chem. 2015 Jan 16;290(3):1496-504. doi: 10.1074/jbc.M114.608281. Epub 2014 Nov 17.


CCR5 Gene Editing of Resting CD4(+) T Cells by Transient ZFN Expression From HIV Envelope Pseudotyped Nonintegrating Lentivirus Confers HIV-1 Resistance in Humanized Mice.

Yi G, Choi JG, Bharaj P, Abraham S, Dang Y, Kafri T, Alozie O, Manjunath MN, Shankar P.

Mol Ther Nucleic Acids. 2014 Sep 30;3:e198. doi: 10.1038/mtna.2014.52.


Quantitative analyses of retinal vascular area and density after different methods to reduce VEGF in a rat model of retinopathy of prematurity.

Wang H, Yang Z, Jiang Y, Flannery J, Hammond S, Kafri T, Vemuri SK, Jones B, Hartnett ME.

Invest Ophthalmol Vis Sci. 2014 Feb 4;55(2):737-44. doi: 10.1167/iovs.13-13429.


Targeting Müller cell-derived VEGF164 to reduce intravitreal neovascularization in the rat model of retinopathy of prematurity.

Jiang Y, Wang H, Culp D, Yang Z, Fotheringham L, Flannery J, Hammond S, Kafri T, Hartnett ME.

Invest Ophthalmol Vis Sci. 2014 Feb 10;55(2):824-31. doi: 10.1167/iovs.13-13755.


Evolutionary etiology of high-grade astrocytomas.

Song Y, Zhang Q, Kutlu B, Difilippantonio S, Bash R, Gilbert D, Yin C, O'Sullivan TN, Yang C, Kozlov S, Bullitt E, McCarthy KD, Kafri T, Louis DN, Miller CR, Hood L, Van Dyke T.

Proc Natl Acad Sci U S A. 2013 Oct 29;110(44):17933-8. doi: 10.1073/pnas.1317026110. Epub 2013 Oct 10.


Short hairpin RNA-mediated knockdown of VEGFA in Müller cells reduces intravitreal neovascularization in a rat model of retinopathy of prematurity.

Wang H, Smith GW, Yang Z, Jiang Y, McCloskey M, Greenberg K, Geisen P, Culp WD, Flannery J, Kafri T, Hammond S, Hartnett ME.

Am J Pathol. 2013 Sep;183(3):964-74.


Integration-deficient lentiviral vectors expressing codon-optimized R338L human FIX restore normal hemostasis in Hemophilia B mice.

Suwanmanee T, Hu G, Gui T, Bartholomae CC, Kutschera I, von Kalle C, Schmidt M, Monahan PE, Kafri T.

Mol Ther. 2014 Mar;22(3):567-574. doi: 10.1038/mt.2013.188. Epub 2013 Aug 14.


Pharmacokinetics and efficacy of PEGylated liposomal doxorubicin in an intracranial model of breast cancer.

Anders CK, Adamo B, Karginova O, Deal AM, Rawal S, Darr D, Schorzman A, Santos C, Bash R, Kafri T, Carey L, Miller CR, Perou CM, Sharpless N, Zamboni WC.

PLoS One. 2013 May 1;8(5):e61359. doi: 10.1371/journal.pone.0061359. Print 2013.


Adeno-associated virus capsid antigen presentation is dependent on endosomal escape.

Li C, He Y, Nicolson S, Hirsch M, Weinberg MS, Zhang P, Kafri T, Samulski RJ.

J Clin Invest. 2013 Mar;123(3):1390-401. doi: 10.1172/JCI66611. Epub 2013 Feb 1.


Dominant-negative androgen receptor inhibition of intracrine androgen-dependent growth of castration-recurrent prostate cancer.

Titus MA, Zeithaml B, Kantor B, Li X, Haack K, Moore DT, Wilson EM, Mohler JL, Kafri T.

PLoS One. 2012;7(1):e30192. doi: 10.1371/journal.pone.0030192. Epub 2012 Jan 17.


The HIV-1 Rev/RRE system is required for HIV-1 5' UTR cis elements to augment encapsidation of heterologous RNA into HIV-1 viral particles.

Cockrell AS, van Praag H, Santistevan N, Ma H, Kafri T.

Retrovirology. 2011 Jun 24;8:51. doi: 10.1186/1742-4690-8-51.


AAV exploits subcellular stress associated with inflammation, endoplasmic reticulum expansion, and misfolded proteins in models of cystic fibrosis.

Johnson JS, Gentzsch M, Zhang L, Ribeiro CM, Kantor B, Kafri T, Pickles RJ, Samulski RJ.

PLoS Pathog. 2011 May;7(5):e1002053. doi: 10.1371/journal.ppat.1002053. Epub 2011 May 19.


Notable reduction in illegitimate integration mediated by a PPT-deleted, nonintegrating lentiviral vector.

Kantor B, Bayer M, Ma H, Samulski J, Li C, McCown T, Kafri T.

Mol Ther. 2011 Mar;19(3):547-56. doi: 10.1038/mt.2010.277. Epub 2010 Dec 14.


Proteasome inhibitors enhance gene delivery by AAV virus vectors expressing large genomes in hemophilia mouse and dog models: a strategy for broad clinical application.

Monahan PE, Lothrop CD, Sun J, Hirsch ML, Kafri T, Kantor B, Sarkar R, Tillson DM, Elia JR, Samulski RJ.

Mol Ther. 2010 Nov;18(11):1907-16. doi: 10.1038/mt.2010.170. Epub 2010 Aug 10.


Regulation of AMPA receptor channels and synaptic plasticity by cofilin phosphatase Slingshot in cortical neurons.

Yuen EY, Liu W, Kafri T, van Praag H, Yan Z.

J Physiol. 2010 Jul 1;588(Pt 13):2361-71. doi: 10.1113/jphysiol.2009.186353. Epub 2010 May 4.


Epigenetic activation of unintegrated HIV-1 genomes by gut-associated short chain fatty acids and its implications for HIV infection.

Kantor B, Ma H, Webster-Cyriaque J, Monahan PE, Kafri T.

Proc Natl Acad Sci U S A. 2009 Nov 3;106(44):18786-91. doi: 10.1073/pnas.0905859106. Epub 2009 Oct 20.


A large U3 deletion causes increased in vivo expression from a nonintegrating lentiviral vector.

Bayer M, Kantor B, Cockrell A, Ma H, Zeithaml B, Li X, McCown T, Kafri T.

Mol Ther. 2008 Dec;16(12):1968-76. doi: 10.1038/mt.2008.199. Epub 2008 Sep 16.


Gene delivery by lentivirus vectors.

Cockrell AS, Kafri T.

Mol Biotechnol. 2007 Jul;36(3):184-204. Review.


Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo.

Li C, Hirsch M, Asokan A, Zeithaml B, Ma H, Kafri T, Samulski RJ.

J Virol. 2007 Jul;81(14):7540-7. Epub 2007 May 2.


A trans-lentiviral packaging cell line for high-titer conditional self-inactivating HIV-1 vectors.

Cockrell AS, Ma H, Fu K, McCown TJ, Kafri T.

Mol Ther. 2006 Aug;14(2):276-84. Epub 2006 Mar 3.


Air-conditioning for regulated transgene expression.

Kafri T.

Gene Ther. 2005 Mar;12(5):383-5. No abstract available.


Transactivator and structurally optimized inducible lentiviral vectors.

Haack K, Cockrell AS, Ma H, Israeli D, Ho SN, McCown TJ, Kafri T.

Mol Ther. 2004 Sep;10(3):585-96.


HIV-1 vectors: fulfillment of expectations, further advancements, and still a way to go.

Cockrell AS, Kafri T.

Curr HIV Res. 2003 Oct;1(4):419-39. Review.


Gene delivery by lentivirus vectors an overview.

Kafri T.

Methods Mol Biol. 2004;246:367-90. Review.


Methylation of HoxA5 and HoxB5 and its relevance to expression during mouse development.

Hershko AY, Kafri T, Fainsod A, Razin A.

Gene. 2003 Jan 2;302(1-2):65-72.


High-level expression of hemoglobin A in human thalassemic erythroid progenitor cells following lentiviral vector delivery of an antisense snRNA.

Vacek MM, Ma H, Gemignani F, Lacerra G, Kafri T, Kole R.

Blood. 2003 Jan 1;101(1):104-11. Epub 2002 Aug 15.


Lentivirus vectors: difficulties and hopes before clinical trials.

Kafri T.

Curr Opin Mol Ther. 2001 Aug;3(4):316-26. Review.


Generation of a stable cell line producing high-titer self-inactivating lentiviral vectors.

Xu K, Ma H, McCown TJ, Verma IM, Kafri T.

Mol Ther. 2001 Jan;3(1):97-104.


Lentiviral vectors: regulated gene expression.

Kafri T, van Praag H, Gage FH, Verma IM.

Mol Ther. 2000 Jun;1(6):516-21.


Integration complexes derived from HIV vectors for rapid assays in vitro.

Hansen MS, Smith GJ 3rd, Kafri T, Molteni V, Siegel JS, Bushman FD.

Nat Biotechnol. 1999 Jun;17(6):578-82.


Gene transfer to human pancreatic endocrine cells using viral vectors.

Leibowitz G, Beattie GM, Kafri T, Cirulli V, Lopez AD, Hayek A, Levine F.

Diabetes. 1999 Apr;48(4):745-53.


Piecing together more efficient gene expression.

Somia NV, Kafri T, Verma IM.

Nat Biotechnol. 1999 Mar;17(3):224-5. No abstract available.


Lentivirus-mediated transduction of islet grafts with interleukin 4 results in sustained gene expression and protection from insulitis.

Gallichan WS, Kafri T, Krahl T, Verma IM, Sarvetnick N.

Hum Gene Ther. 1998 Dec 10;9(18):2717-26.


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