Medical College of Wisconsin
CTSICores SearchResearch InformaticsREDCap

Publications indexed to the term Genetic Therapy

1Lentivirus-mediated gene therapy for Fabry disease. (Khan A, Barber DL, Huang J, Rupar CA, Rip JW, Auray-Blais C, Boutin M, O'Hoski P, Gargulak K, McKillop WM, Fraser G, Wasim S, LeMoine K, Jelinski S, Chaudhry A, Prokopishyn N, Morel CF, Couban S, Duggan PR, Fowler DH, Keating A, West ML, Foley R, Medin JA) Nat Commun 2021 02 25;12(1):1178    
1Platelet-Targeted FVIII Gene Therapy Restores Hemostasis and Induces Immune Tolerance for Hemophilia A. (Cai Y, Shi Q) Front Immunol 2020;11:964    
1A Comparison of Inducible Gene Expression Platforms: Implications for Recombinant Adeno-Associated Virus (rAAV) Vector-Mediated Ocular Gene Therapy. (Lipinski DM) Adv Exp Med Biol 2019;1185:79-83       2 Citations
1AAV-Mediated Gene Transfer Restores a Normal Muscle Transcriptome in a Canine Model of X-Linked Myotubular Myopathy. (Dupont JB, Guo J, Renaud-Gabardos E, Poulard K, Latournerie V, Lawlor MW, Grange RW, Gray JT, Buj-Bello A, Childers MK, Mack DL) Mol Ther 2020 02 05;28(2):382-393       5 Citations
2Nongenotoxic antibody-drug conjugate conditioning enables safe and effective platelet gene therapy of hemophilia A mice. (Gao C, Schroeder JA, Xue F, Jing W, Cai Y, Scheck A, Subramaniam S, Rao S, Weiler H, Czechowicz A, Shi Q) Blood Adv 2019 09 24;3(18):2700-2711       13 Citations
2AAV-encoded CaV2.2 peptide aptamer CBD3A6K for primary sensory neuron-targeted treatment of established neuropathic pain. (Yu H, Shin SM, Xiang H, Chao D, Cai Y, Xu H, Khanna R, Pan B, Hogan QH) Gene Ther 2019 08;26(7-8):308-323       7 Citations
1An update on gene therapy for lysosomal storage disorders. (Nagree MS, Scalia S, McKillop WM, Medin JA) Expert Opin Biol Ther 2019 07;19(7):655-670       9 Citations
1Lentiviral interleukin-10 gene therapy: Safety and questions. (Liu M, Juvet SC, Medin JA, Martinu T, Oishi H, Keshavjee S) J Thorac Cardiovasc Surg 2019 02;157(2):818-819    
2The impact of GPIbα on platelet-targeted FVIII gene therapy in hemophilia A mice with pre-existing anti-FVIII immunity. (Chen J, Schroeder JA, Luo X, Montgomery RR, Shi Q) J Thromb Haemost 2019 03;17(3):449-459       7 Citations
1Engineered Thymidine-Active Deoxycytidine Kinase for Bystander Killing of Malignant Cells. (Neschadim A, Medin JA) Methods Mol Biol 2019;1895:149-163    
1A Destabilizing Domain Allows for Fast, Noninvasive, Conditional Control of Protein Abundance in the Mouse Eye - Implications for Ocular Gene Therapy. (Datta S, Renwick M, Chau VQ, Zhang F, Nettesheim ER, Lipinski DM, Hulleman JD) Invest Ophthalmol Vis Sci 2018 10 01;59(12):4909-4920       7 Citations
3Current status and strategies of long noncoding RNA research for diabetic cardiomyopathy. (Pant T, Dhanasekaran A, Fang J, Bai X, Bosnjak ZJ, Liang M, Ge ZD) BMC Cardiovasc Disord 2018 10 20;18(1):197       14 Citations
4Platelet Gene Therapy Promotes Targeted Peripheral Tolerance by Clonal Deletion and Induction of Antigen-Specific Regulatory T Cells. (Luo X, Chen J, Schroeder JA, Allen KP, Baumgartner CK, Malarkannan S, Hu J, Williams CB, Shi Q) Front Immunol 2018;9:1950       8 Citations
1Pathological Issues in Dystrophinopathy in the Age of Genetic Therapies. (Shahnoor N, Siebers EM, Brown KJ, Lawlor MW) Annu Rev Pathol 2019 01 24;14:105-126       1 Citation
1Development of an inducible anti-VEGF rAAV gene therapy strategy for the treatment of wet AMD. (Reid CA, Nettesheim ER, Connor TB, Lipinski DM) Sci Rep 2018 08 06;8(1):11763       14 Citations
1Brain-Derived Neurotrophic Factor as a Treatment Option for Retinal Degeneration. (Daly C, Ward R, Reynolds AL, Galvin O, Collery RF, Kennedy BN) Adv Exp Med Biol 2018;1074:465-471       6 Citations
1Novel Combination BMP7 and HGF Gene Therapy Instigates Selective Myofibroblast Apoptosis and Reduces Corneal Haze In Vivo. (Gupta S, Fink MK, Ghosh A, Tripathi R, Sinha PR, Sharma A, Hesemann NP, Chaurasia SS, Giuliano EA, Mohan RR) Invest Ophthalmol Vis Sci 2018 02 01;59(2):1045-1057       23 Citations
2Dextran Enhances the Lentiviral Transduction Efficiency of Murine and Human Primary NK Cells. (Nanbakhsh A, Best B, Riese M, Rao S, Wang L, Medin J, Thakar MS, Malarkannan S) J Vis Exp 2018 01 15(131)       3 Citations
1Improvement of Photoreceptor Targeting via Intravitreal Delivery in Mouse and Human Retina Using Combinatory rAAV2 Capsid Mutant Vectors. (Reid CA, Ertel KJ, Lipinski DM) Invest Ophthalmol Vis Sci 2017 12 01;58(14):6429-6439       14 Citations
1Small and Micro-Scale Recombinant Adeno-Associated Virus Production and Purification for Ocular Gene Therapy Applications. (Reid CA, Lipinski DM) Methods Mol Biol 2018;1715:19-31       4 Citations
1A phase I trial of intraperitoneal GEN-1, an IL-12 plasmid formulated with PEG-PEI-cholesterol lipopolymer, administered with pegylated liposomal doxorubicin in patients with recurrent or persistent epithelial ovarian, fallopian tube or primary peritoneal cancers: An NRG Oncology/Gynecologic Oncology Group study. (Thaker PH, Brady WE, Lankes HA, Odunsi K, Bradley WH, Moore KN, Muller CY, Anwer K, Schilder RJ, Alvarez RD, Fracasso PM) Gynecol Oncol 2017 11;147(2):283-290       13 Citations
1Immune tolerance induced by platelet-targeted factor VIII gene therapy in hemophilia A mice is CD4 T cell mediated. (Chen Y, Luo X, Schroeder JA, Chen J, Baumgartner CK, Hu J, Shi Q) J Thromb Haemost 2017 10;15(10):1994-2004       18 Citations
1Practical considerations for chimeric antigen receptor design and delivery. (Oldham RAA, Medin JA) Expert Opin Biol Ther 2017 08;17(8):961-978       9 Citations
1Long-term effects of systemic gene therapy in a canine model of myotubular myopathy. (Elverman M, Goddard MA, Mack D, Snyder JM, Lawlor MW, Meng H, Beggs AH, Buj-Bello A, Poulard K, Marsh AP, Grange RW, Kelly VE, Childers MK) Muscle Nerve 2017 Nov;56(5):943-953       29 Citations
1Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs. (Mack DL, Poulard K, Goddard MA, Latournerie V, Snyder JM, Grange RW, Elverman MR, Denard J, Veron P, Buscara L, Le Bec C, Hogrel JY, Brezovec AG, Meng H, Yang L, Liu F, O'Callaghan M, Gopal N, Kelly VE, Smith BK, Strande JL, Mavilio F, Beggs AH, Mingozzi F, Lawlor MW, Buj-Bello A, Childers MK) Mol Ther 2017 04 05;25(4):839-854       50 Citations
1Combined chondroitinase and KLF7 expression reduce net retraction of sensory and CST axons from sites of spinal injury. (Wang Z, Winsor K, Nienhaus C, Hess E, Blackmore MG) Neurobiol Dis 2017 Mar;99:24-35       21 Citations
1Single residue AAV capsid mutation improves transduction of photoreceptors in the Abca4-/- mouse and bipolar cells in the rd1 mouse and human retina ex vivo. (De Silva SR, Charbel Issa P, Singh MS, Lipinski DM, Barnea-Cramer AO, Walker NJ, Barnard AR, Hankins MW, MacLaren RE) Gene Ther 2016 11;23(11):767-774       15 Citations
3Targeting factor VIII expression to platelets for hemophilia A gene therapy does not induce an apparent thrombotic risk in mice. (Baumgartner CK, Mattson JG, Weiler H, Shi Q, Montgomery RR) J Thromb Haemost 2017 01;15(1):98-109       10 Citations
1A miR-192-EGR1-HOXB9 regulatory network controls the angiogenic switch in cancer. (Wu SY, Rupaimoole R, Shen F, Pradeep S, Pecot CV, Ivan C, Nagaraja AS, Gharpure KM, Pham E, Hatakeyama H, McGuire MH, Haemmerle M, Vidal-Anaya V, Olsen C, Rodriguez-Aguayo C, Filant J, Ehsanipour EA, Herbrich SM, Maiti SN, Huang L, Kim JH, Zhang X, Han HD, Armaiz-Pena GN, Seviour EG, Tucker S, Zhang M, Yang D, Cooper LJ, Ali-Fehmi R, Bar-Eli M, Lee JS, Ram PT, Baggerly KA, Lopez-Berestein G, Hung MC, Sood AK) Nat Commun 2016 Apr 04;7:11169       72 Citations
1Megakaryocyte- and megakaryocyte precursor-related gene therapies. (Wilcox DA) Blood 2016 Mar 10;127(10):1260-8       8 Citations
2Emerging immunotherapy in pediatric lymphoma. (Erker C, Harker-Murray P, Burke MJ) Future Oncol 2016 Jan;12(2):257-70       3 Citations
2AAV-Mediated Gene Transfer to Dorsal Root Ganglion. (Yu H, Fischer G, Hogan QH) Methods Mol Biol 2016;1382:251-61       8 Citations
2Perhexiline activates KLF14 and reduces atherosclerosis by modulating ApoA-I production. (Guo Y, Fan Y, Zhang J, Lomberk GA, Zhou Z, Sun L, Mathison AJ, Garcia-Barrio MT, Zhang J, Zeng L, Li L, Pennathur S, Willer CJ, Rader DJ, Urrutia R, Chen YE) J Clin Invest 2015 Oct 01;125(10):3819-30       40 Citations
1Systemic Vascular Transduction by Capsid Mutant Adeno-Associated Virus After Intravenous Injection. (Lipinski DM, Reid CA, Boye SL, Peterson JJ, Qi X, Boye SE, Boulton ME, Hauswirth WW) Hum Gene Ther 2015 Nov;26(11):767-76       6 Citations
1Evaluation of Bystander Cell Killing Effects in Suicide Gene Therapy of Cancer: Engineered Thymidylate Kinase (TMPK)/AZT Enzyme-Prodrug Axis. (Sato T, Neschadim A, Nakagawa R, Yanagisawa T, Medin JA) Methods Mol Biol 2015;1317:55-67       2 Citations
2The important role of von Willebrand factor in platelet-derived FVIII gene therapy for murine hemophilia A in the presence of inhibitory antibodies. (Shi Q, Schroeder JA, Kuether EL, Montgomery RR) J Thromb Haemost 2015 Jul;13(7):1301-9       15 Citations
1CNTF Gene Therapy Confers Lifelong Neuroprotection in a Mouse Model of Human Retinitis Pigmentosa. (Lipinski DM, Barnard AR, Singh MS, Martin C, Lee EJ, Davies WIL, MacLaren RE) Mol Ther 2015 Aug;23(8):1308-1319       43 Citations
1Blocking the adhesion cascade at the premetastatic niche for prevention of breast cancer metastasis. (Kang SA, Hasan N, Mann AP, Zheng W, Zhao L, Morris L, Zhu W, Zhao YD, Suh KS, Dooley WC, Volk D, Gorenstein DG, Cristofanilli M, Rui H, Tanaka T) Mol Ther 2015 Jun;23(6):1044-1054       30 Citations
1MASH1/Ascl1a leads to GAP43 expression and axon regeneration in the adult CNS. (Williams RR, Venkatesh I, Pearse DD, Udvadia AJ, Bunge MB) PLoS One 2015;10(3):e0118918       27 Citations
1Smoothing the crescent curve: sickle cell disease. (Talano JA, Cairo MS) Hematology Am Soc Hematol Educ Program 2014 Dec 05;2014(1):468-74       3 Citations
1Advances in sickle cell therapies in the hydroxyurea era. (Field JJ, Nathan DG) Mol Med 2014 Dec 16;20 Suppl 1:S37-42       12 Citations
2Blood and marrow transplant clinical trials network state of the Science Symposium 2014. (Appelbaum FR, Anasetti C, Antin JH, Atkins H, Davies S, Devine S, Giralt S, Heslop H, Laport G, Lee SJ, Logan B, Pasquini M, Pulsipher M, Stadtmauer E, Wingard JR, Horowitz MM) Biol Blood Marrow Transplant 2015 Feb;21(2):202-24       14 Citations
2Non-myeloablative conditioning with busulfan before hematopoietic stem cell transplantation leads to phenotypic correction of murine Bernard-Soulier syndrome. (Kanaji S, Fahs SA, Ware J, Montgomery RR, Shi Q) J Thromb Haemost 2014 Oct;12(10):1726-32       6 Citations
1Delayed administration of a bio-engineered zinc-finger VEGF-A gene therapy is neuroprotective and attenuates allodynia following traumatic spinal cord injury. (Figley SA, Liu Y, Karadimas SK, Satkunendrarajah K, Fettes P, Spratt SK, Lee G, Ando D, Surosky R, Giedlin M, Fehlings MG) PLoS One 2014;9(5):e96137       21 Citations
1Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy. (Childers MK, Joubert R, Poulard K, Moal C, Grange RW, Doering JA, Lawlor MW, Rider BE, Jamet T, Danièle N, Martin S, Rivière C, Soker T, Hammer C, Van Wittenberghe L, Lockard M, Guan X, Goddard M, Mitchell E, Barber J, Williams JK, Mack DL, Furth ME, Vignaud A, Masurier C, Mavilio F, Moullier P, Beggs AH, Buj-Bello A) Sci Transl Med 2014 Jan 22;6(220):220ra10       89 Citations
2Platelet gene therapy corrects the hemophilic phenotype in immunocompromised hemophilia A mice transplanted with genetically manipulated human cord blood stem cells. (Shi Q, Kuether EL, Chen Y, Schroeder JA, Fahs SA, Montgomery RR) Blood 2014 Jan 16;123(3):395-403       32 Citations
3Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A. (Du LM, Nurden P, Nurden AT, Nichols TC, Bellinger DA, Jensen ES, Haberichter SL, Merricks E, Raymer RA, Fang J, Koukouritaki SB, Jacobi PM, Hawkins TB, Cornetta K, Shi Q, Wilcox DA) Nat Commun 2013;4:2773       71 Citations
1The engineered thymidylate kinase (TMPK)/AZT enzyme-prodrug axis offers efficient bystander cell killing for suicide gene therapy of cancer. (Sato T, Neschadim A, Lavie A, Yanagisawa T, Medin JA) PLoS One 2013;8(10):e78711       21 Citations
1Retinal structure and function in achromatopsia: implications for gene therapy. (Sundaram V, Wilde C, Aboshiha J, Cowing J, Han C, Langlo CS, Chana R, Davidson AE, Sergouniotis PI, Bainbridge JW, Ali RR, Dubra A, Rubin G, Webster AR, Moore AT, Nardini M, Carroll J, Michaelides M) Ophthalmology 2014 Jan;121(1):234-245       89 Citations
1Human cone visual pigment deletions spare sufficient photoreceptors to warrant gene therapy. (Cideciyan AV, Hufnagel RB, Carroll J, Sumaroka A, Luo X, Schwartz SB, Dubra A, Land M, Michaelides M, Gardner JC, Hardcastle AJ, Moore AT, Sisk RA, Ahmed ZM, Kohl S, Wissinger B, Jacobson SG) Hum Gene Ther 2013 Dec;24(12):993-1006       59 Citations
1Platelet gene therapy by lentiviral gene delivery to hematopoietic stem cells restores hemostasis and induces humoral immune tolerance in FIX(null) mice. (Chen Y, Schroeder JA, Kuether EL, Zhang G, Shi Q) Mol Ther 2014 Jan;22(1):169-77       39 Citations
1Glanzmann thrombasthenia: state of the art and future directions. (Nurden AT, Pillois X, Wilcox DA) Semin Thromb Hemost 2013 Sep;39(6):642-55       62 Citations
1Harnessing autophagy for cell fate control gene therapy. (Felizardo TC, Foley J, Steed K, Dropulic B, Amarnath S, Medin JA, Fowler DH) Autophagy 2013 Jul;9(7):1069-79       5 Citations
1Lentivirus IL-10 gene therapy down-regulates IL-17 and attenuates mouse orthotopic lung allograft rejection. (Hirayama S, Sato M, Loisel-Meyer S, Matsuda Y, Oishi H, Guan Z, Saito T, Yeung J, Cypel M, Hwang DM, Medin JA, Liu M, Keshavjee S) Am J Transplant 2013 Jun;13(6):1586-93       31 Citations
1Liver transplantation for lethal genetic syndromes: a novel model of personalized genomic medicine. (Petrowsky H, Brunicardi FC, Leow VM, Venick RS, Agopian V, Kaldas FM, Zarrinpar A, Markovic D, McDiarmid SV, Hong JC, Farmer DG, Hiatt JR, Busuttil RW) J Am Coll Surg 2013 Apr;216(4):534-43; discussion 543-4       6 Citations
1Clinical applications of retinal gene therapy. (Lipinski DM, Thake M, MacLaren RE) Prog Retin Eye Res 2013 Jan;32:22-47       78 Citations
1Gene therapy for aromatic L-amino acid decarboxylase deficiency. (Zwagerman NT, Richardson RM) Neurosurgery 2012 Oct;71(4):N10-2       4 Citations
1Pre-mRNA splicing in disease and therapeutics. (Singh RK, Cooper TA) Trends Mol Med 2012 Aug;18(8):472-82       271 Citations
1Preclinical efficacy of the oncolytic measles virus expressing the sodium iodide symporter in iodine non-avid anaplastic thyroid cancer: a novel therapeutic agent allowing noninvasive imaging and radioiodine therapy. (Reddi HV, Madde P, McDonough SJ, Trujillo MA, Morris JC 3rd, Myers RM, Peng KW, Russell SJ, McIver B, Eberhardt NL) Cancer Gene Ther 2012 Sep;19(9):659-65       28 Citations
3Lentivirus-mediated platelet gene therapy of murine hemophilia A with pre-existing anti-factor VIII immunity. (Kuether EL, Schroeder JA, Fahs SA, Cooley BC, Chen Y, Montgomery RR, Wilcox DA, Shi Q) J Thromb Haemost 2012 Aug;10(8):1570-80       79 Citations
1Lentivector transduction improves outcomes over transplantation of human HSCs alone in NOD/SCID/Fabry mice. (Pacienza N, Yoshimitsu M, Mizue N, Au BC, Wang JC, Fan X, Takenaka T, Medin JA) Mol Ther 2012 Jul;20(7):1454-61       14 Citations
2Platelet and endothelial expression of clotting factors for the treatment of hemophilia. (Montgomery RR, Shi Q) Thromb Res 2012 May;129 Suppl 2:S46-8       11 Citations
1Regulation of acute graft-versus-host disease by microRNA-155. (Ranganathan P, Heaphy CE, Costinean S, Stauffer N, Na C, Hamadani M, Santhanam R, Mao C, Taylor PA, Sandhu S, He G, Shana'ah A, Nuovo GJ, Lagana A, Cascione L, Obad S, Broom O, Kauppinen S, Byrd JC, Caligiuri M, Perrotti D, Hadley GA, Marcucci G, Devine SM, Blazar BR, Croce CM, Garzon R) Blood 2012 May 17;119(20):4786-97       102 Citations
1Bystander killing of malignant cells via the delivery of engineered thymidine-active deoxycytidine kinase for suicide gene therapy of cancer. (Neschadim A, Wang JC, Lavie A, Medin JA) Cancer Gene Ther 2012 May;19(5):320-7       18 Citations
1Cell fate control gene therapy based on engineered variants of human deoxycytidine kinase. (Neschadim A, Wang JC, Sato T, Fowler DH, Lavie A, Medin JA) Mol Ther 2012 May;20(5):1002-13       15 Citations
2Correction of murine Bernard-Soulier syndrome by lentivirus-mediated gene therapy. (Kanaji S, Kuether EL, Fahs SA, Schroeder JA, Ware J, Montgomery RR, Shi Q) Mol Ther 2012 Mar;20(3):625-32       15 Citations
1Optimization of hCFTR lung expression in mice using DNA nanoparticles. (Padegimas L, Kowalczyk TH, Adams S, Gedeon CR, Oette SM, Dines K, Hyatt SL, Sesenoglu-Laird O, Tyr O, Moen RC, Cooper MJ) Mol Ther 2012 Jan;20(1):63-72    
1Targeted nonviral gene-based inhibition of Gα(i/o)-mediated vagal signaling in the posterior left atrium decreases vagal-induced atrial fibrillation. (Aistrup GL, Cokic I, Ng J, Gordon D, Koduri H, Browne S, Arapi D, Segon Y, Goldstein J, Angulo A, Wasserstrom JA, Goldberger JJ, Kadish AH, Arora R) Heart Rhythm 2011 Nov;8(11):1722-9       31 Citations
2Platelet gene therapy improves hemostatic function for integrin alphaIIbbeta3-deficient dogs. (Fang J, Jensen ES, Boudreaux MK, Du LM, Hawkins TB, Koukouritaki SB, Cornetta K, Wilcox DA) Proc Natl Acad Sci U S A 2011 Jun 07;108(23):9583-8       41 Citations
1Direct injection into the dorsal root ganglion: technical, behavioral, and histological observations. (Fischer G, Kostic S, Nakai H, Park F, Sapunar D, Yu H, Hogan Q) J Neurosci Methods 2011 Jul 15;199(1):43-55       49 Citations
1Cystic fibrosis transmembrane conductance regulator with a shortened R domain rescues the intestinal phenotype of CFTR-/- mice. (Ostedgaard LS, Meyerholz DK, Vermeer DW, Karp PH, Schneider L, Sigmund CD, Welsh MJ) Proc Natl Acad Sci U S A 2011 Feb 15;108(7):2921-6       12 Citations
1Autologous transplantation of lentivector/acid ceramidase-transduced hematopoietic cells in nonhuman primates. (Walia JS, Neschadim A, Lopez-Perez O, Alayoubi A, Fan X, Carpentier S, Madden M, Lee CJ, Cheung F, Jaffray DA, Levade T, McCart JA, Medin JA) Hum Gene Ther 2011 Jun;22(6):679-87       22 Citations
2Alternative strategies for gene therapy of hemophilia. (Montgomery RR, Shi Q) Hematology Am Soc Hematol Educ Program 2010;2010:197-202       5 Citations
1Promoter-specific lentivectors for long-term, cardiac-directed therapy of Fabry disease. (Lee CJ, Fan X, Guo X, Medin JA) J Cardiol 2011 Jan;57(1):115-22       29 Citations
1Hemophilia: an amazing 35-year journey from the depths of HIV to the threshold of cure. (White GC) Trans Am Clin Climatol Assoc 2010;121:61-73; discussion 74-5       14 Citations
1Unique strategies for therapeutic gene transfer in haemophilia A and haemophilia BWFH State-of-the-Art Session on Therapeutic Gene Transfer Buenos Aires, Argentina. (Montgomery RR, Monahan PE, Ozelo MC) Haemophilia 2010 Jul;16 Suppl 5:29-34       6 Citations
2Platelets as delivery systems for disease treatments. (Shi Q, Montgomery RR) Adv Drug Deliv Rev 2010 Sep 30;62(12):1196-203       29 Citations
1Alpha-galactosidase A-Tat fusion enhances storage reduction in hearts and kidneys of Fabry mice. (Higuchi K, Yoshimitsu M, Fan X, Guo X, Rasaiah VI, Yen J, Tei C, Takenaka T, Medin JA) Mol Med 2010 May-Jun;16(5-6):216-21       10 Citations
2Factor IX ectopically expressed in platelets can be stored in alpha-granules and corrects the phenotype of hemophilia B mice. (Zhang G, Shi Q, Fahs SA, Kuether EL, Walsh CE, Montgomery RR) Blood 2010 Aug 26;116(8):1235-43       36 Citations
1Ex vivo delivery of GDNF maintains motor function and prevents neuronal loss in a transgenic mouse model of Huntington's disease. (Ebert AD, Barber AE, Heins BM, Svendsen CN) Exp Neurol 2010 Jul;224(1):155-62       59 Citations
1Sequencing and characterization of the porcine α-galactosidase A gene: towards the generation of a porcine model for Fabry disease. (Yoshimitsu M, Higuchi K, Fan X, Takao S, Medin JA, Tei C, Takenaka T) Mol Biol Rep 2011 Jun;38(5):3145-52       1 Citation
1Gene therapy for red-green colour blindness in adult primates. (Mancuso K, Hauswirth WW, Li Q, Connor TB, Kuchenbecker JA, Mauck MC, Neitz J, Neitz M) Nature 2009 Oct 08;461(7265):784-7       212 Citations
1Novel application of lentiviral vectors towards treatment of graft-versus-host disease. (Scaife MD, Neschadim A, Fowler DH, Medin JA) Expert Opin Biol Ther 2009 Jun;9(6):749-61       6 Citations
1Potent induction of B- and T-cell immunity against human carcinoembryonic antigen-expressing tumors in human carcinoembryonic antigen transgenic mice mediated by direct lentivector injection. (Loisel-Meyer S, Felizardo T, Mariotti J, Mossoba ME, Foley JE, Kammerer R, Mizue N, Keefe R, McCart JA, Zimmermann W, Dropulic B, Fowler DH, Medin JA) Mol Cancer Ther 2009 Mar;8(3):692-702       14 Citations
2The sunset of somatic genetics and the dawn of epigenetics: a new frontier in pancreatic cancer research. (Lomberk G, Mathison AJ, Grzenda A, Urrutia R) Curr Opin Gastroenterol 2008 Sep;24(5):597-602       18 Citations
1Transient anti-CD40L co-stimulation blockade prevents immune responses against human bullous pemphigoid antigen 2: implications for gene therapy. (Lanschuetzer CM, Olasz EB, Lazarova Z, Yancey KB) J Invest Dermatol 2009 May;129(5):1203-7       13 Citations
1In vivo delivery of human acid ceramidase via cord blood transplantation and direct injection of lentivirus as novel treatment approaches for Farber disease. (Ramsubir S, Nonaka T, Girbés CB, Carpentier S, Levade T, Medin JA) Mol Genet Metab 2008 Nov;95(3):133-41       21 Citations
1Polymeric gene delivery of ischemia-inducible VEGF significantly attenuates infarct size and apoptosis following myocardial infarct. (Yockman JW, Choi D, Whitten MG, Chang CW, Kastenmeier A, Erickson H, Albanil A, Lee M, Kim SW, Bull DA) Gene Ther 2009 Jan;16(1):127-35       50 Citations
1Novel polymer carriers and gene constructs for treatment of myocardial ischemia and infarction. (Yockman JW, Kastenmeier A, Erickson HM, Brumbach JG, Whitten MG, Albanil A, Li DY, Kim SW, Bull DA) J Control Release 2008 Dec 18;132(3):260-6       47 Citations
1IL-12 immunotherapy of murine leukaemia: comparison of systemic versus gene modified cell therapy. (Labbe A, Nelles M, Walia J, Jia L, Furlonger C, Nonaka T, Medin JA, Paige CJ) J Cell Mol Med 2009 Aug;13(8B):1962-1976       15 Citations
1Immuno-gene therapy approaches for cancer: from in vitro studies to clinical trials. (Loisel-Meyer S, Foley R, Medin JA) Front Biosci 2008 May 01;13:3202-14       19 Citations
4Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity. (Shi Q, Fahs SA, Wilcox DA, Kuether EL, Morateck PA, Mareno N, Weiler H, Montgomery RR) Blood 2008 Oct 01;112(7):2713-21       73 Citations
1von Willebrand disease (VWD): evidence-based diagnosis and management guidelines, the National Heart, Lung, and Blood Institute (NHLBI) Expert Panel report (USA). (Nichols WL, Hultin MB, James AH, Manco-Johnson MJ, Montgomery RR, Ortel TL, Rick ME, Sadler JE, Weinstein M, Yawn BP) Haemophilia 2008 Mar;14(2):171-232    
1Cardiac overexpression of angiotensin converting enzyme 2 protects the heart from ischemia-induced pathophysiology. (Der Sarkissian S, Grobe JL, Yuan L, Narielwala DR, Walter GA, Katovich MJ, Raizada MK) Hypertension 2008 Mar;51(3):712-8       113 Citations
1Tumor protection following vaccination with low doses of lentivirally transduced DCs expressing the self-antigen erbB2. (Mossoba ME, Walia JS, Rasaiah VI, Buxhoeveden N, Head R, Ying C, Foley JE, Bramson JL, Fowler DH, Medin JA) Mol Ther 2008 Mar;16(3):607-17       22 Citations
1Predicting preferential DNA vector insertion sites: implications for functional genomics and gene therapy. (Hackett CS, Geurts AM, Hackett PB) Genome Biol 2007;8 Suppl 1:S12       49 Citations
1A roadmap to safe, efficient, and stable lentivirus-mediated gene therapy with hematopoietic cell transplantation. (Neschadim A, McCart JA, Keating A, Medin JA) Biol Blood Marrow Transplant 2007 Dec;13(12):1407-16       33 Citations
1Downregulation of Sirt1 by antisense oligonucleotides induces apoptosis and enhances radiation sensitization in A549 lung cancer cells. (Sun Y, Sun D, Li F, Tian L, Li C, Li L, Lin R, Wang S) Lung Cancer 2007 Oct;58(1):21-9       62 Citations
1Results from a phase II multicenter, double-blind placebo-controlled study of Del-1 (VLTS-589) for intermittent claudication in subjects with peripheral arterial disease. (Grossman PM, Mendelsohn F, Henry TD, Hermiller JB, Litt M, Saucedo JF, Weiss RJ, Kandzari DE, Kleiman N, Anderson RD, Gottlieb D, Karlsberg R, Snell J, Rocha-Singh K) Am Heart J 2007 May;153(5):874-80       68 Citations
1Lentiviral vector-mediated genetic modification of human neural progenitor cells for ex vivo gene therapy. (Capowski EE, Schneider BL, Ebert AD, Seehus CR, Szulc J, Zufferey R, Aebischer P, Svendsen CN) J Neurosci Methods 2007 Jul 30;163(2):338-49       62 Citations
1Anti-CD25 targeted killing of bicistronically transduced cells: a novel safety mechanism against retroviral genotoxicity. (Ramsubir S, Yoshimitsu M, Medin JA) Mol Ther 2007 Jun;15(6):1174-81       8 Citations
1Engineered human tmpk/AZT as a novel enzyme/prodrug axis for suicide gene therapy. (Sato T, Neschadim A, Konrad M, Fowler DH, Lavie A, Medin JA) Mol Ther 2007 May;15(5):962-70       72 Citations
1Lentiviral vectors for gene therapy of heart disease. (Higuchi K, Medin JA) J Cardiol 2007 Jan;49(1):1-11       3 Citations
4Lentivirus-mediated platelet-derived factor VIII gene therapy in murine haemophilia A. (Shi Q, Wilcox DA, Fahs SA, Fang J, Johnson BD, DU LM, Desai D, Montgomery RR) J Thromb Haemost 2007 Feb;5(2):352-61       99 Citations
1Multiple reduced-intensity conditioning regimens facilitate correction of Fabry mice after transplantation of transduced cells. (Liang SB, Yoshimitsu M, Poeppl A, Rasaiah VI, Cai J, Fowler DH, Medin JA) Mol Ther 2007 Mar;15(3):618-27       11 Citations
1Correction of cardiac abnormalities in fabry mice by direct intraventricular injection of a recombinant lentiviral vector that engineers expression of alpha-galactosidase A. (Yoshimitsu M, Higuchi K, Dawood F, Rasaiah VI, Ayach B, Chen M, Liu P, Medin JA) Circ J 2006 Nov;70(11):1503-8       30 Citations
1Combination of PTEN gene therapy and radiation inhibits the growth of human prostate cancer xenografts. (Anai S, Goodison S, Shiverick K, Iczkowski K, Tanaka M, Rosser CJ) Hum Gene Ther 2006 Oct;17(10):975-84       36 Citations
1Efficient correction of Fabry mice and patient cells mediated by lentiviral transduction of hematopoietic stem/progenitor cells. (Yoshimitsu M, Higuchi K, Ramsubir S, Nonaka T, Rasaiah VI, Siatskas C, Liang SB, Murray GJ, Brady RO, Medin JA) Gene Ther 2007 Feb;14(3):256-65       37 Citations
1Gene transfer of pigment epithelium-derived factor suppresses tumor growth and angiogenesis in a hepatoblastoma xenograft model. (Browne M, Stellmach V, Cornwell M, Chung C, Doll JA, Lee EJ, Jameson JL, Reynolds M, Superina RA, Abramson LP, Crawford SE) Pediatr Res 2006 Sep;60(3):282-7       22 Citations
4Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies. (Shi Q, Wilcox DA, Fahs SA, Weiler H, Wells CW, Cooley BC, Desai D, Morateck PA, Gorski J, Montgomery RR) J Clin Invest 2006 Jul;116(7):1974-82       130 Citations
1Functional expression of mouse relaxin and mouse relaxin-3 in the lung from an Ebola virus glycoprotein-pseudotyped lentivirus via tracheal delivery. (Silvertown JD, Walia JS, Summerlee AJ, Medin JA) Endocrinology 2006 Aug;147(8):3797-808       22 Citations
1Human neural progenitors deliver glial cell line-derived neurotrophic factor to parkinsonian rodents and aged primates. (Behrstock S, Ebert A, McHugh J, Vosberg S, Moore J, Schneider B, Capowski E, Hei D, Kordower J, Aebischer P, Svendsen CN) Gene Ther 2006 Mar;13(5):379-88       135 Citations
1A new tool in the battle against Alzheimer's disease and aging: ex vivo gene therapy. (Ebert AD, Svendsen CN) Rejuvenation Res 2005;8(3):131-4       19 Citations
1Searching for obesity genes: progress and prospects. (Liu YJ, Xiao P, Xiong DH, Recker RR, Deng HW) Drugs Today (Barc) 2005 May;41(5):345-62       8 Citations
1Specific pharmacological dimerization of KDR in lentivirally transduced human hematopoietic cells activates anti-apoptotic and proliferative mechanisms. (Siatskas C, Underwood J, Ramezani A, Hawley RG, Medin JA) FASEB J 2005 Oct;19(12):1752-4       9 Citations
3Therapeutic expression of the platelet-specific integrin, alphaIIbbeta3, in a murine model for Glanzmann thrombasthenia. (Fang J, Hodivala-Dilke K, Johnson BD, Du LM, Hynes RO, White GC 2nd, Wilcox DA) Blood 2005 Oct 15;106(8):2671-9       66 Citations
1Gene therapy for hemophilia? (White GC 2nd, Roberts HR) J Thromb Haemost 2005 Jun;3(6):1312       4 Citations
1Learning from failure: congestive heart failure in the postgenomic age. (Benjamin IJ, Schneider MD) J Clin Invest 2005 Mar;115(3):495-9       55 Citations
1Efficient transfer of PSA and PSMA cDNAs into DCs generates antibody and T cell antitumor responses in vivo. (Medin JA, Liang SB, Hou JW, Kelley LS, Peace DJ, Fowler DH) Cancer Gene Ther 2005 Jun;12(6):540-51       24 Citations
1Angiotensin-converting enzyme 2 as a novel target for gene therapy for hypertension. (Katovich MJ, Grobe JL, Huentelman M, Raizada MK) Exp Physiol 2005 May;90(3):299-305       28 Citations
3Targeting platelet GPIbalpha transgene expression to human megakaryocytes and forming a complete complex with endogenous GPIbbeta and GPIX. (Shi Q, Wilcox DA, Morateck PA, Fahs SA, Kenny D, Montgomery RR) J Thromb Haemost 2004 Nov;2(11):1989-97       31 Citations
1Metafectene is superior to lipofectamine in the transfection of G(s) alpha prostate cancer cells. (Iczkowski KA, Omara-Opyene AL, Klösel R) Mol Biotechnol 2004 Oct;28(2):97-103       7 Citations
1Infectivity enhanced adenoviral-mediated mda-7/IL-24 gene therapy for ovarian carcinoma. (Leath CA 3rd, Kataram M, Bhagavatula P, Gopalkrishnan RV, Dent P, Fisher PB, Pereboev A, Carey D, Lebedeva IV, Haisma HJ, Alvarez RD, Curiel DT, Mahasreshti PJ) Gynecol Oncol 2004 Aug;94(2):352-62       25 Citations
1Autologous vascular smooth muscle cell-based myocardial gene therapy to induce coronary collateral growth. (Hattan N, Warltier D, Gu W, Kolz C, Chilian WM, Weihrauch D) Am J Physiol Heart Circ Physiol 2004 Aug;287(2):H488-93       23 Citations
5Induction of megakaryocytes to synthesize and store a releasable pool of human factor VIII. (Wilcox DA, Shi Q, Nurden P, Haberichter SL, Rosenberg JB, Johnson BD, Nurden AT, White GC 2nd, Montgomery RR) J Thromb Haemost 2003 Dec;1(12):2477-89       46 Citations
2Gene therapy for platelet disorders: studies with Glanzmann's thrombasthenia. (Wilcox DA, White GC 2nd) J Thromb Haemost 2003 Nov;1(11):2300-11       25 Citations
3Factor VIII ectopically expressed in platelets: efficacy in hemophilia A treatment. (Yarovoi HV, Kufrin D, Eslin DE, Thornton MA, Haberichter SL, Shi Q, Zhu H, Camire R, Fakharzadeh SS, Kowalska MA, Wilcox DA, Sachais BS, Montgomery RR, Poncz M) Blood 2003 Dec 01;102(12):4006-13       119 Citations
1Gene transfer establishes primacy of striated vs. smooth muscle sarcoglycan complex in limb-girdle muscular dystrophy. (Durbeej M, Sawatzki SM, Barresi R, Schmainda KM, Allamand V, Michele DE, Campbell KP) Proc Natl Acad Sci U S A 2003 Jul 22;100(15):8910-5       30 Citations
1Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion. (Powell JS, Ragni MV, White GC 2nd, Lusher JM, Hillman-Wiseman C, Moon TE, Cole V, Ramanathan-Girish S, Roehl H, Sajjadi N, Jolly DJ, Hurst D) Blood 2003 Sep 15;102(6):2038-45       161 Citations
2Inhibition of transcription factor Stat5 induces cell death of human prostate cancer cells. (Ahonen TJ, Xie J, LeBaron MJ, Zhu J, Nurmi M, Alanen K, Rui H, Nevalainen MT) J Biol Chem 2003 Jul 18;278(29):27287-92       104 Citations
1Radiation increases the activity of oncolytic adenovirus cancer gene therapy vectors that overexpress the ADP (E3-11.6K) protein. (Toth K, Tarakanova V, Doronin K, Ward P, Kuppuswamy M, Locke JE, Dawson JE, Kim HJ, Wold WS) Cancer Gene Ther 2003 Mar;10(3):193-200       39 Citations
1Hemophilia gene therapy: update. (Monahan PE, White GC 2nd) Curr Opin Hematol 2002 Sep;9(5):430-6       8 Citations
1Molecular advances in genetic skin diseases. (Siegel DH, Howard R) Curr Opin Pediatr 2002 Aug;14(4):419-25       11 Citations
1Post-transduction events in retrovirus-mediated gene therapy involving hematopoietic stem cells: beyond efficiency issues. (Medin JA, Fowler DH) J Cell Biochem Suppl 2002;38:46-54       4 Citations
1Meeting report: Physiological Genomics of Cardiovascular Disease: from Technology to Physiology. (Glueck SB, Sigmund CD) Physiol Genomics 2002;9(3):135-6       1 Citation
1Lower blood pressure in floxed angiotensinogen mice after adenoviral delivery of Cre-recombinase. (Stec DE, Keen HL, Sigmund CD) Hypertension 2002 Feb;39(2 Pt 2):629-33       23 Citations
1Gene therapy for Fabry disease. (Siatskas C, Medin JA) J Inherit Metab Dis 2001;24 Suppl 2:25-41; discussion 11-2       31 Citations
1Gene transfer in the cardiovascular system: update 2000. (Medin JA, Buttrick PM) Heart Dis 2000 Nov-Dec;2(6):409-21       2 Citations
2Can progression of autosomal dominant or autosomal recessive polycystic kidney disease be prevented? (Davis ID, MacRae Dell K, Sweeney WE, Avner ED) Semin Nephrol 2001 Sep;21(5):430-40       27 Citations
1Multimodality management of localized pancreatic cancer. (Wayne JD, Wolff RA, Pisters PW, Evans DB) Cancer J 2001 Jul-Aug;7 Suppl 1:S35-46       12 Citations
1Gene therapy in hemophilia: clinical trials update. (White GC 2nd) Thromb Haemost 2001 Jul;86(1):172-7       27 Citations
1K cells: a novel target for insulin gene therapy for the prevention of diabetes. (Corbett JA) Trends Endocrinol Metab 2001 May-Jun;12(4):140-2       11 Citations
1Preselective gene therapy for Fabry disease. (Qin G, Takenaka T, Telsch K, Kelley L, Howard T, Levade T, Deans R, Howard BH, Malech HL, Brady RO, Medin JA) Proc Natl Acad Sci U S A 2001 Mar 13;98(6):3428-33       64 Citations
1Brown Norway chromosome 13 confers protection from high salt to consomic Dahl S rat. (Cowley AW Jr, Roman RJ, Kaldunski ML, Dumas P, Dickhout JG, Greene AS, Jacob HJ) Hypertension 2001 Feb;37(2 Pt 2):456-61       164 Citations
1Biomarker distribution after injection into the canine prostate: implications for gene therapy. (Siemens DR, Iwasawa T, Austin JC, Williams RD, See WA, Hedican SP, Tartaglia J, Flynn CM, Cohen MB, Rodgers J, Ratliff TL) BJU Int 2000 Dec;86(9):1076-83       4 Citations
1Genetic induction of a releasable pool of factor VIII in human endothelial cells. (Rosenberg JB, Greengard JS, Montgomery RR) Arterioscler Thromb Vasc Biol 2000 Dec;20(12):2689-95       65 Citations
1Early adenovirus-mediated gene transfer effectively prevents muscular dystrophy in alpha-sarcoglycan-deficient mice. (Allamand V, Donahue KM, Straub V, Davisson RL, Davidson BL, Campbell KP) Gene Ther 2000 Aug;7(16):1385-91       36 Citations
1Gene therapy for hemophilia: a step closer to reality. (White GC 2nd, Roberts HR) Mol Ther 2000 Mar;1(3):207-8       4 Citations
1Interactions between von Willebrand factor and Factor VIII: where did they first meet. (Montgomery RR, Gill JC) J Pediatr Hematol Oncol 2000 May-Jun;22(3):269-75       33 Citations
2Megakaryocyte-targeted synthesis of the integrin beta(3)-subunit results in the phenotypic correction of Glanzmann thrombasthenia. (Wilcox DA, Olsen JC, Ishizawa L, Bray PF, French DL, Steeber DA, Bell WR, Griffith M, White GC 2nd) Blood 2000 Jun 15;95(12):3645-51       44 Citations
1Current approaches and future strategies for pancreatic carcinoma. (Wolff RA, Chiao P, Lenzi R, Pisters PW, Lee JE, Janjan NA, Crane CH, Evans DB, Abbruzzese JL) Invest New Drugs 2000 Feb;18(1):43-56       35 Citations
1Intracoronary adenovirus-mediated delivery and overexpression of the beta(2)-adrenergic receptor in the heart : prospects for molecular ventricular assistance. (Shah AS, Lilly RE, Kypson AP, Tai O, Hata JA, Pippen A, Silvestry SC, Lefkowitz RJ, Glower DD, Koch WJ) Circulation 2000 Feb 01;101(4):408-14       109 Citations
2Cellular pathophysiology of cystic kidney disease: insight into future therapies. (Avner ED, Woychik RP, Dell KM, Sweeney WE) Int J Dev Biol 1999;43(5):457-61       12 Citations
1Circulating alpha-galactosidase A derived from transduced bone marrow cells: relevance for corrective gene transfer for Fabry disease. (Takenaka T, Qin G, Brady RO, Medin JA) Hum Gene Ther 1999 Aug 10;10(12):1931-9       36 Citations
2Integrin alphaIIb promoter-targeted expression of gene products in megakaryocytes derived from retrovirus-transduced human hematopoietic cells. (Wilcox DA, Olsen JC, Ishizawa L, Griffith M, White GC 2nd) Proc Natl Acad Sci U S A 1999 Aug 17;96(17):9654-9       47 Citations
1Enzymatic and functional correction along with long-term enzyme secretion from transduced bone marrow hematopoietic stem/progenitor and stromal cells derived from patients with Fabry disease. (Takenaka T, Hendrickson CS, Tworek DM, Tudor M, Schiffmann R, Brady RO, Medin JA) Exp Hematol 1999 Jul;27(7):1149-59       32 Citations
1Ex vivo expansion and genetic marking of primitive human and baboon hematopoietic cells. (Medin JA, Brandt JE, Rozler E, Nelson M, Bartholomew A, Li C, Turian J, Chute J, Chung T, Hoffman R) Ann N Y Acad Sci 1999 Apr 30;872:233-40; discussion 240-2       6 Citations
1In vivo gene therapy with interleukin-12 inhibits primary vascular tumor growth and induces apoptosis in a mouse model. (Wang C, Quevedo ME, Lannutti BJ, Gordon KB, Guo D, Sun W, Paller AS) J Invest Dermatol 1999 May;112(5):775-81       32 Citations
1Adenovirus-mediated wild-type p53 tumor suppressor gene therapy induces apoptosis and suppresses growth of human pancreatic cancer [seecomments]. (Bouvet M, Bold RJ, Lee J, Evans DB, Abbruzzese JL, Chiao PJ, McConkey DJ, Chandra J, Chada S, Fang B, Roth JA) Ann Surg Oncol 1998 Dec;5(8):681-8       113 Citations
1Gene therapy and pancreatic cancer. (Pearson AS, Bouvet M, Evans DB, Roth JA) Front Biosci 1998 Nov 01;3:E230-7       25 Citations
1Future therapies for the treatment of bladder neoplasms. (See WA, Berman CJ) Semin Surg Oncol 1997 Sep-Oct;13(5):376-88       1 Citation
1Selection of retrovirally transduced cells to enhance the efficiency of gene therapy. (Medin JA, Karlsson S) Proc Assoc Am Physicians 1997 Mar;109(2):111-9       11 Citations
1Viral vectors for gene therapy of hematopoietic cells. (Medin JA, Karlsson S) Immunotechnology 1997 Mar;3(1):3-19       20 Citations
1Complexes of non-cationic liposomes and histone H1 mediate efficient transfection of DNA without encapsulation. (Hagstrom JE, Sebestyen MG, Budker V, Ludtke JJ, Fritz JD, Wolff JA) Biochim Biophys Acta 1996 Oct 02;1284(1):47-55       71 Citations
1Transient protection of human T-cells from human immunodeficiency virus type 1 infection by transduction with adeno-associated viral vectors which express RNA decoys. (Smith C, Lee SW, Wong E, Gallardo H, Page K, Gaspar O, Lebkowski J, Gilboa E) Antiviral Res 1996 Oct;32(2):99-115       18 Citations
2Functional correction of renal defects in a mouse model for ARPKD through expression of the cloned wild-type Tg737 cDNA. (Yoder BK, Richards WG, Sommardahl C, Sweeney WE, Michaud EJ, Wilkinson JE, Avner ED, Woychik RP) Kidney Int 1996 Oct;50(4):1240-8       32 Citations
1Correction in trans for Fabry disease: expression, secretion and uptake of alpha-galactosidase A in patient-derived cells driven by a high-titer recombinant retroviral vector. (Medin JA, Tudor M, Simovitch R, Quirk JM, Jacobson S, Murray GJ, Brady RO) Proc Natl Acad Sci U S A 1996 Jul 23;93(15):7917-22       59 Citations
1The in vivo quantification of myocardial performance in rabbits: a model for evaluation of cardiac gene therapy. (Silvestry SC, Taylor DA, Lilly RE, Atkins BZ, Marathe US, Davis JW, Kraus W, Glower DD) J Mol Cell Cardiol 1996 May;28(5):815-23       14 Citations
1A bicistronic therapeutic retroviral vector enables sorting of transduced CD34+ cells and corrects the enzyme deficiency in cells from Gaucher patients. (Medin JA, Migita M, Pawliuk R, Jacobson S, Amiri M, Kluepfel-Stahl S, Brady RO, Humphries RK, Karlsson S) Blood 1996 Mar 01;87(5):1754-62       34 Citations
1Selection of transduced CD34+ progenitors and enzymatic correction of cells from Gaucher patients, with bicistronic vectors. (Migita M, Medin JA, Pawliuk R, Jacobson S, Nagle JW, Anderson S, Amiri M, Humphries RK, Karlsson S) Proc Natl Acad Sci U S A 1995 Dec 19;92(26):12075-9       50 Citations
1Treatment of the hemophilias. (Moll S, White GC 2nd) Curr Opin Hematol 1995 Sep;2(5):386-94       2 Citations
1Expression of deletion-containing dystrophins in mdx muscle: implications for gene therapy and dystrophin function. (Fritz JD, Danko I, Roberds SL, Campbell KP, Latendresse JS, Wolff JA) Pediatr Res 1995 Jun;37(6):693-700       10 Citations
1Toward gene therapy for Niemann-Pick disease (NPD): separation of retrovirally corrected and noncorrected NPD fibroblasts using a novel fluorescent sphingomyelin. (Dinur T, Schuchman EH, Fibach E, Dagan A, Suchi M, Desnick RJ, Gatt S) Hum Gene Ther 1992 Dec;3(6):633-9       15 Citations
1Retroviral-mediated transfer of the human acid sphingomyelinase cDNA: correction of the metabolic defect in cultured Niemann-Pick disease cells. (Suchi M, Dinur T, Desnick RJ, Gatt S, Pereira L, Gilboa E, Schuchman EH) Proc Natl Acad Sci U S A 1992 Apr 15;89(8):3227-31       29 Citations