Miguel Sena-Esteves, PhD

2016

Vodicka P, Chase K, Iuliano M, Valentine DT, Sapp E, Lu B, Kegel-Gleason KB, Sena-Esteves M, Aronin N, DiFiglia M. Effects of Exogenous NUB1 Expression in the Striatum of HDQ175/Q7 Mice. J Huntingtons Dis. 2016 Jun 13;5(2):163-74. doi:10.3233/JHD-160195. PubMed PMID: 27314618.  

GuhaSarkar D, Su Q, Gao G, Sena-Esteves M. Systemic AAV9-IFNβ gene delivery treats highly invasive glioblastoma. Neuro Oncol. 2016 May 18. pii: now097. [Epubahead of print] PubMed PMID: 27194146.

Choudhury SR, Fitzpatrick Z, Harris AF, Maitland SA, Ferreira JS, Zhang Y, Ma S, Sharma RB, Gray-Edwards HL, Johnson JA, Johnson AK, Alonso LC, Punzo C, Wagner KR, Maguire CA, Kotin RM, Martin DR, Sena-Esteves M. In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy. Mol Ther. 2016 Jun 7. doi: 10.1038/mt.2016.84. [Epub ahead of print] PubMed PMID: 27117222. 

Choudhury SR, Hudry E, Maguire CA, Sena-Esteves M, Breakefield XO, Grandi P. Viral vectors for therapy of neurologic diseases. Neuropharmacology. 2016 Feb 21.pii: S0028-3908(16)30048-X. doi: 10.1016/j.neuropharm.2016.02.013. [Epub ahead of print] Review. PubMed PMID: 26905292. 

Stoica L, Todeasa SH, Cabrera GT, Salameh JS, ElMallah MK, Mueller C, Brown RH Jr, Sena-Esteves M. Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model. Ann Neurol. 2016 Apr;79(4):687-700. doi: 10.1002/ana.24618. Epub 2016 Mar 11. PubMed PMID: 26891182. 

Choudhury SR, Harris AF, Cabral DJ, Keeler AM, Sapp E, Ferreira JS, Gray-Edwards HL, Johnson JA, Johnson AK, Su Q, Stoica L, DiFiglia M, Aronin N, Martin DR, Gao G, Sena-Esteves M. Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector. Mol Ther. 2016 Apr;24(4):726-35. doi: 10.1038/mt.2015.231. Epub 2015 Dec 28. PubMed PMID: 26708003; PubMed Central PMCID: PMC4886933.

2015

Prabhakar S, Zhang X, Goto J, Han S, Lai C, Bronson R, Sena-Esteves M, Ramesh V, Stemmer-Rachamimov A, Kwiatkowski DJ, Breakefield XO. Survival benefit and phenotypic improvement by hamartin gene therapy in a tuberous sclerosis mouse brain model. Neurobiol Dis. 2015 Oct;82:22-31. doi: 10.1016/j.nbd.2015.04.018. Epub 2015 May 24. PubMed PMID: 26019056.

Gray-Edwards HL, Brunson BL, Holland M, Hespel AM, Bradbury AM, McCurdy VJ, Beadlescomb PM, Randle AN, Salibi N, Denney TS, Beyers RJ, Johnson AK, Voyles ML, Montgomery RD, Wilson DU, Hudson JA, Cox NR, Baker HJ, Sena-Esteves M, Martin DR. Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy. Mol Genet Metab. 2015 Sep-Oct;116(1-2):80-7. doi: 10.1016/j.ymgme.2015.05.003. Epub 2015 May 8. PubMed PMID: 25971245. 

Weismann CM, Ferreira J, Keeler AM, Su Q, Qui L, Shaffer SA, Xu Z, Gao G,Sena-Esteves M. Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan. Hum Mol Genet. 2015 Aug 1;24(15):4353-64. doi: 10.1093/hmg/ddv168. Epub 2015 May 10. PubMed PMID: 25964428; PubMed Central PMCID: PMC4492398.

Rockwell HE, McCurdy VJ, Eaton SC, Wilson DU, Johnson AK, Randle AN, Bradbury AM, Gray-Edwards HL, Baker HJ, Hudson JA, Cox NR, Sena-Esteves M, Seyfried TN, Martin DR. AAV-mediated gene delivery in a feline model of Sandhoff disease corrects lysosomal storage in the central nervous system. ASN Neuro. 2015 Apr 13;7(2). pii: 1759091415569908. doi: 10.1177/1759091415569908. Print 2015 Mar-Apr. PubMed PMID: 25873306; PubMed Central PMCID: PMC4720176.

Akgoc Z, Sena-Esteves M, Martin DR, Han X, d'Azzo A, Seyfried TN. Bis(monoacylglycero)phosphate: a secondary storage lipid in the gangliosidoses. J Lipid Res. 2015 May;56(5):1006-13. doi: 10.1194/jlr.M057851. Epub 2015 Mar 20. PubMed PMID: 25795792; PubMed Central PMCID: PMC4409277.

McCurdy VJ, Rockwell HE, Arthur JR, Bradbury AM, Johnson AK, Randle AN, Brunson BL, Hwang M, Gray-Edwards HL, Morrison NE, Johnson JA, Baker HJ, Cox NR, Seyfried TN, Sena-Esteves M, Martin DR. Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease. Gene Ther. 2015 Feb;22(2):181-9. doi: 10.1038/gt.2014.108. Epub 2014 Dec PubMed PMID: 25474439. 

Bradbury AM, Gray-Edwards HL, Shirley JL, McCurdy VJ, Colaco AN, Randle AN, Christopherson PW, Bird AC, Johnson AK, Wilson DU, Hudson JA, De Pompa NL, Sorjonen DC, Brunson BL, Jeyakumar M, Platt FM, Baker HJ, Cox NR, Sena-Esteves M, Martin DR. Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff disease. Exp Neurol. 2015 Jan;263:102-12. doi: 10.1016/j.expneurol.2014.09.020. Epub 2014 Oct 5. PubMed PMID: 25284324; PubMed Central PMCID: PMC4262540. 

2014

Batista AR, Gianni D, Ventosa M, Coelho AV, Almeida MR, Sena-Esteves M, Saraiva MJ. Gene therapy approach to FAP: in vivo influence of T119M in TTR deposition in a transgenic V30M mouse model. Gene Ther. 2014 Dec;21(12):1041-50. doi: 10.1038/gt.2014.86. Epub 2014 Oct 2. Erratum in: Gene Ther. 2014 Dec;21(12):1065. PubMed PMID: 25273354. 

Maguire CA, Ramirez SH, Merkel SF, Sena-Esteves M, Breakefield XO. Gene therapy for the nervous system: challenges and new strategies. Neurotherapeutics. 2014 Oct;11(4):817-39. doi: 10.1007/s13311-014-0299-5. Review. PubMed PMID: 25159276; PubMed Central PMCID: PMC4391389. 

Seyfried TN, Rockwell HE, Heinecke KA, Martin DR, Sena-Esteves M. Ganglioside storage diseases: on the road to management. Adv Neurobiol. 2014;9:485-99. doi: 10.1007/978-1-4939-1154-7_22. PubMed PMID: 25151393.

O'Reilly M, Federoff HJ, Fong Y, Kohn DB, Patterson AP, Ahmed N, Asokan A, Boye SE, Crystal RG, De Oliveira S, Gargiulo L, Harper SQ, Ikeda Y, Jambou R, Montgomery M, Prograis L, Rosenthal E, Sterman DH, Vandenberghe LH, Zoloth L, Abedi M, Adair J, Adusumilli PS, Goins WF, Gray J, Monahan P, Popplewell L, Sena-Esteves M, Tannous B, Weber T, Wierda W, Gopal-Srivastava R, McDonald CL, Rosenblum D, Corrigan-Curay J. Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013. Hum Gene Ther. 2014 Jun;25(6):488-97. doi: 10.1089/hum.2014.045. Review. PubMed PMID: 24773122; PubMed Central PMCID: PMC4064731. 

McCurdy VJ, Johnson AK, Gray-Edwards HL, Randle AN, Brunson BL, Morrison NE, Salibi N, Johnson JA, Hwang M, Beyers RJ, Leroy SG, Maitland S, Denney TS, Cox NR, Baker HJ, Sena-Esteves M, Martin DR. Sustained normalization of neurological disease after intracranial gene therapy in a feline model. Sci Transl Med. 2014 Apr 9;6(231):231ra48. doi: 10.1126/scitranslmed.3007733. PubMed PMID: 24718858; PubMed Central PMCID: PMC4412602. 

Cordeira JW, Felsted JA, Teillon S, Daftary S, Panessiti M, Wirth J, Sena-Esteves M, Rios M. Hypothalamic dysfunction of the thrombospondin receptor α2δ-1 underlies the overeating and obesity triggered by brain-derived neurotrophic factor deficiency. J Neurosci. 2014 Jan 8;34(2):554-65. doi: 10.1523/JNEUROSCI.1572-13.2014. PubMed PMID: 24403154; PubMed Central PMCID: PMC3870936. 

2013

Bienemann K, Staege MS, Howe SJ, Sena-Esteves M, Hanenberg H, Kramm CM. Targeted expression of human folylpolyglutamate synthase for selective enhancement of methotrexate chemotherapy in osteosarcoma cells. Cancer Gene Ther. 2013 Sep;20(9):514-20. doi: 10.1038/cgt.2013.48. Epub 2013 Aug 16. PubMed PMID: 23949282. 

Maguire CA, Bovenberg MS, Crommentuijn MH, Niers JM, Kerami M, Teng J, Sena-Esteves M, Badr CE, Tannous BA. Triple bioluminescence imaging for in vivo monitoring of cellular processes. Mol Ther Nucleic Acids. 2013 Jun 18;2:e99. doi: 10.1038/mtna.2013.25. PubMed PMID: 23778500; PubMed Central PMCID: PMC3696905. 

Prabhakar S, Goto J, Zhang X, Sena-Esteves M, Bronson R, Brockmann J, Gianni D, Wojtkiewicz GR, Chen JW, Stemmer-Rachamimov A, Kwiatkowski DJ, Breakefield XO. Stochastic model of Tsc1 lesions in mouse brain. PLoS One. 2013 May 16;8(5):e64224. doi: 10.1371/journal.pone.0064224. Print 2013. Erratum in: PLoS One. 2013;8(11). doi:10.1371/annotation/6a5b0a50-27e4-49bc-b82a-9267dd63af53. Zuang, Xuan [corrected to Zhang, Xuan]. PubMed PMID: 23696872; PubMed Central PMCID: PMC3655945. 

Bradbury AM, Cochran JN, McCurdy VJ, Johnson AK, Brunson BL, Gray-Edwards H, Leroy SG, Hwang M, Randle AN, Jackson LS, Morrison NE, Baek RC, Seyfried TN, Cheng SH, Cox NR, Baker HJ, Cachón-González MB, Cox TM, Sena-Esteves M, Martin DR. Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy. Mol Ther. 2013 Jul;21(7):1306-15. doi: 10.1038/mt.2013.86. Epub 2013 May 21. PubMed PMID: 23689599; PubMed Central PMCID: PMC3702100. 

Stoica L, Ahmed SS, Gao G, Sena-Esteves M. Gene transfer to the CNS using recombinant adeno-associated virus. Curr Protoc Microbiol. 2013;Chapter 14:Unit14D.5. doi: 10.1002/9780471729259.mc14d05s29. PubMed PMID: 23686825; PubMed Central PMCID: PMC4135440. 

Batista AR, Sena-Esteves M, Saraiva MJ. Hepatic production of transthyretin L12P leads to intracellular lysosomal aggregates in a new somatic transgenic mouse model. Biochim Biophys Acta. 2013 Aug;1832(8):1183-93. doi: 10.1016/j.bbadis.2013.04.001. Epub 2013 Apr 8. PubMed PMID: 23579071. 

de Vrij J, Maas SL, van Nispen M, Sena-Esteves M, Limpens RW, Koster AJ, Leenstra S, Lamfers ML, Broekman ML. Quantification of nanosized extracellular membrane vesicles with scanning ion occlusion sensing. Nanomedicine (Lond). 2013 Sep;8(9):1443-58. doi: 10.2217/nnm.12.173. Epub 2013 Feb 5. PubMed PMID: 23384702. 

van der Bom IM, Moser RP, Gao G, Mondo E, O'Connell D, Gounis MJ, McGowan S, Chaurette J, Bishop N, Sena-Esteves MS, Mueller C, Aronin N. Finding the striatum in sheep: use of a multi-modal guided approach for convection enhanced delivery. J Huntingtons Dis. 2013;2(1):41-5. doi: 10.3233/JHD-130053. PubMed PMID: 25063428. 

Prabhakar S, Taherian M, Gianni D, Conlon TJ, Fulci G, Brockmann J, Stemmer-Rachamimov A, Sena-Esteves M, Breakefield XO, Brenner GJ. Regression of schwannomas induced by adeno-associated virus-mediated delivery of caspase-1. Hum Gene Ther. 2013 Feb;24(2):152-62. doi: 10.1089/hum.2012.094. Epub 2013 Jan 30. PubMed PMID: 23140466; PubMed Central PMCID: PMC3581065.

2012

Almeida S, Zhang Z, Coppola G, Mao W, Futai K, Karydas A, Geschwind MD, Tartaglia MC, Gao F, Gianni D, Sena-Esteves M, Geschwind DH, Miller BL, Farese RV Jr, Gao FB. Induced pluripotent stem cell models of progranulin-deficient frontotemporal dementia uncover specific reversible neuronal defects. Cell Rep. 2012 Oct 25;2(4):789-98. doi: 10.1016/j.celrep.2012.09.007. Epub 2012 Oct 11. PubMed PMID: 23063362; PubMed Central PMCID: PMC3532907. 

Maguire CA, Balaj L, Sivaraman S, Crommentuijn MH, Ericsson M, Mincheva-Nilsson L, Baranov V, Gianni D, Tannous BA, Sena-Esteves M, Breakefield XO, Skog J. Microvesicle-associated AAV vector as a novel gene delivery system. Mol Ther. 2012 May;20(5):960-71. doi: 10.1038/mt.2011.303. Epub 2012 Feb 7. PubMed PMID: 22314290; PubMed Central PMCID: PMC3345986.

van der Bom IM, Moser RP, Gao G, Sena-Esteves M, Aronin N, Gounis MJ. Frameless multimodal image guidance of localized convection-enhanced delivery of therapeutics in the brain. J Neurointerv Surg. 2013 Jan 1;5(1):69-72. doi: 10.1136/neurintsurg-2011-010170. Epub 2011 Dec 22. PubMed PMID: 22193239; PubMed Central PMCID: PMC3533401. 

Bruinstroop E, Cano G, Vanderhorst VG, Cavalcante JC, Wirth J, Sena-Esteves M, Saper CB. Spinal projections of the A5, A6 (locus coeruleus), and A7 noradrenergic cell groups in rats. J Comp Neurol. 2012 Jun 15;520(9):1985-2001. doi: 10.1002/cne.23024. PubMed PMID: 22173709; PubMed Central PMCID: PMC3508755.

Pike LS, Tannous BA, Deliolanis NC, Hsich G, Morse D, Tung CH, Sena-Esteves M, Breakefield XO. Imaging gene delivery in a mouse model of congenital neuronal ceroid lipofuscinosis. Gene Ther. 2011 Dec;18(12):1173-8. doi: 10.1038/gt.2011.118. Epub 2011 Sep 8. Erratum in: Gene Ther. 2012 Nov;19(11):1121. PubMed PMID: 21900963; PubMed Central PMCID: PMC3235265. 

2011

Zhang H, Yang B, Mu X, Ahmed SS, Su Q, He R, Wang H, Mueller C, Sena-Esteves M, Brown R, Xu Z, Gao G. Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system. Mol Ther. 2011 Aug;19(8):1440-8. doi: 10.1038/mt.2011.98. Epub 2011 May 24. PubMed PMID: 21610699; PubMed Central PMCID: PMC3149178. 

Bowers WJ, Breakefield XO, Sena-Esteves M. Genetic therapy for the nervous system. Hum Mol Genet. 2011 Apr 15;20(R1):R28-41. doi: 10.1093/hmg/ddr110. Epub 2011 Mar 23. Review. PubMed PMID: 21429918; PubMed Central PMCID: PMC3095060. 

2010

Kegel KB, Sapp E, Alexander J, Reeves P, Bleckmann D, Sobin L, Masso N, Valencia A, Jeong H, Krainc D, Palacino J, Curtis D, Kuhn R, Betschart C, Sena-Esteves M, Aronin N, Paganetti P, Difiglia M. Huntingtin cleavage product A forms in neurons and is reduced by gamma-secretase inhibitors. Mol Neurodegener. 2010 Dec 14;5:58. doi: 10.1186/1750-1326-5-58. PubMed PMID: 21156064; PubMed Central PMCID: PMC3018386. 

Baek RC, Broekman ML, Leroy SG, Tierney LA, Sandberg MA, d'Azzo A, Seyfried TN, Sena-Esteves M. AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival. PLoS One. 2010 Oct 18;5(10):e13468. doi: 10.1371/journal.pone.0013468. PubMed PMID: 20976108; PubMed Central PMCID: PMC2956705. 

Breakefield XO, Sena-Esteves M. Healing genes in the nervous system. Neuron. 2010 Oct 21;68(2):178-81. doi: 10.1016/j.neuron.2010.10.005. PubMed PMID: 20955923; PubMed Central PMCID: PMC3076203.

White E, Bienemann A, Sena-Esteves M, Taylor H, Bunnun C, Castrique E, Gill S. Evaluation and optimization of the administration of recombinant adeno-associated viral vectors (serotypes 2/1, 2/2, 2/rh8, 2/9, and 2/rh10) by convection-enhanced delivery to the striatum. Hum Gene Ther. 2011 Feb;22(2):237-51. doi: 10.1089/hum.2010.129. Epub 2011 Feb 2. PubMed PMID: 20836701. 

Giove TJ, Sena-Esteves M, Eldred WD. Transduction of the inner mouse retina using AAVrh8 and AAVrh10 via intravitreal injection. Exp Eye Res. 2010 Nov;91(5):652-9. doi: 10.1016/j.exer.2010.08.011. Epub 2010 Aug 17. PubMed PMID: 20723541; PubMed Central PMCID: PMC2962726. 

Grandi P, Fernandez J, Szentirmai O, Carter R, Gianni D, Sena-Esteves M, Breakefield XO. Targeting HSV-1 virions for specific binding to epidermal growth factor receptor-vIII-bearing tumor cells. Cancer Gene Ther. 2010 Sep;17(9):655-63. doi: 10.1038/cgt.2010.22. Epub 2010 May 28. PubMed PMID: 20508670; PubMed Central PMCID: PMC2923688.

Cordeira JW, Frank L, Sena-Esteves M, Pothos EN, Rios M. Brain-derived neurotrophic factor regulates hedonic feeding by acting on the mesolimbic dopamine system. J Neurosci. 2010 Feb 17;30(7):2533-41. doi: 10.1523/JNEUROSCI.5768-09.2010. PubMed PMID: 20164338; PubMed Central PMCID: PMC2846779.

Prabhakar S, Brenner GJ, Sung B, Messerli SM, Mao J, Sena-Esteves M, Stemmer-Rachamimov A, Tannous B, Breakefield XO. Imaging and therapy of experimental schwannomas using HSV amplicon vector-encoding apoptotic protein under Schwann cell promoter. Cancer Gene Ther. 2010 Apr;17(4):266-74. doi: 10.1038/cgt.2009.71. Epub 2009 Oct 16. PubMed PMID: 19834516; PubMed Central PMCID: PMC2857743. 

Maguire CA, Gianni D, Meijer DH, Shaket LA, Wakimoto H, Rabkin SD, Gao G, Sena-Esteves M. Directed evolution of adeno-associated virus for glioma cell transduction. J Neurooncol. 2010 Feb;96(3):337-47. doi: 10.1007/s11060-009-9972-7. Epub 2009 Jul 19. PubMed PMID: 19618115; PubMed Central PMCID: PMC2892971.

2009

Maguire CA, Deliolanis NC, Pike L, Niers JM, Tjon-Kon-Fat LA, Sena-Esteves M, Tannous BA. Gaussia luciferase variant for high-throughput functional screening applications. Anal Chem. 2009 Aug 15;81(16):7102-6. doi: 10.1021/ac901234r. PubMed PMID: 19601604; PubMed Central PMCID: PMC2846205. 

Tannous BA, Christensen AP, Pike L, Wurdinger T, Perry KF, Saydam O, Jacobs AH, García-Añoveros J, Weissleder R, Sena-Esteves M, Corey DP, Breakefield XO. Mutant sodium channel for tumor therapy. Mol Ther. 2009 May;17(5):810-9. doi: 10.1038/mt.2009.33. Epub 2009 Mar 3. PubMed PMID: 19259066; PubMed Central PMCID: PMC2751883.

Meijer DH, Maguire CA, LeRoy SG, Sena-Esteves M. Controlling brain tumor growth by intraventricular administration of an AAV vector encoding IFN-beta. Cancer Gene Ther. 2009 Aug;16(8):664-71. doi: 10.1038/cgt.2009.8. Epub 2009 Feb PubMed PMID: 19197327; PubMed Central PMCID: PMC2946391. 

Skog J, Würdinger T, van Rijn S, Meijer DH, Gainche L, Sena-Esteves M, Curry WT Jr, Carter BS, Krichevsky AM, Breakefield XO. Glioblastoma microvesicles transport RNA and proteins that promote tumour growth and provide diagnostic biomarkers. Nat Cell Biol. 2008 Dec;10(12):1470-6. doi: 10.1038/ncb1800. Epub 2008 Nov 16. PubMed PMID: 19011622; PubMed Central PMCID: PMC3423894. 

Broekman ML, Tierney LA, Benn C, Chawla P, Cha JH, Sena-Esteves M. Mechanisms of distribution of mouse beta-galactosidase in the adult GM1-gangliosidosis brain. Gene Ther. 2009 Feb;16(2):303-8. doi: 10.1038/gt.2008.149. Epub 2008 Sep 25. PubMed PMID: 18818671. 

2008

Maguire CA, Meijer DH, LeRoy SG, Tierney LA, Broekman ML, Costa FF, Breakefield XO, Stemmer-Rachamimov A, Sena-Esteves M. Preventing growth of brain tumors by creating a zone of resistance. Mol Ther. 2008 Oct;16(10):1695-702. doi: 10.1038/mt.2008.168. Epub 2008 Aug 19. PubMed PMID: 18714312; PubMed Central PMCID: PMC2863297. 

2007

Unger TJ, Calderon GA, Bradley LC, Sena-Esteves M, Rios M. Selective deletion of Bdnf in the ventromedial and dorsomedial hypothalamus of adult mice results in hyperphagic behavior and obesity. J Neurosci. 2007 Dec 26;27(52):14265-74. PubMed PMID: 18160634. 

DiFiglia M, Sena-Esteves M, Chase K, Sapp E, Pfister E, Sass M, Yoder J, Reeves P, Pandey RK, Rajeev KG, Manoharan M, Sah DW, Zamore PD, Aronin N. Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits. Proc Natl Acad Sci U S A. 2007 Oct 23;104(43):17204-9. Epub 2007 Oct 16. PubMed PMID: 17940007; PubMed Central PMCID: PMC2040405.

Lin IC, Slemp AE, Hwang C, Sena-Esteves M, Nah HD, Kirschner RE. Dihydrotestosterone stimulates proliferation and differentiation of fetal calvarial osteoblasts and dural cells and induces cranial suture fusion. Plast Reconstr Surg. 2007 Oct;120(5):1137-47. PubMed PMID: 17898587. 

Winkeler A, Sena-Esteves M, Paulis LE, Li H, Waerzeggers Y, Rückriem B, Himmelreich U, Klein M, Monfared P, Rueger MA, Heneka M, Vollmar S, Hoehn M, Fraefel C, Graf R, Wienhard K, Heiss WD, Jacobs AH. Switching on the lights for gene therapy. PLoS One. 2007 Jun 13;2(6):e528. PubMed PMID: 17565381; PubMed Central PMCID: PMC1885827.

Tesco G, Koh YH, Kang EL, Cameron AN, Das S, Sena-Esteves M, Hiltunen M, Yang SH, Zhong Z, Shen Y, Simpkins JW, Tanzi RE. Depletion of GGA3 stabilizes BACE and enhances beta-secretase activity. Neuron. 2007 Jun 7;54(5):721-37. PubMed PMID: 17553422; PubMed Central PMCID: PMC1973166.

Oehmig A, Cortés ML, Perry KF, Sena-Esteves M, Fraefel C, Breakefield XO. Integration of active human beta-galactosidase gene (100 kb) into genome using HSV/AAV amplicon vector. Gene Ther. 2007 Jul;14(14):1078-91. Epub 2007 Apr 26. PubMed PMID: 17460718. 

Broekman ML, Baek RC, Comer LA, Fernandez JL, Seyfried TN, Sena-Esteves M. Complete correction of enzymatic deficiency and neurochemistry in the GM1-gangliosidosis mouse brain by neonatal adeno-associated virus-mediated gene delivery. Mol Ther. 2007 Jan;15(1):30-7. PubMed PMID: 17164772.

2006

Broekman ML, Comer LA, Hyman BT, Sena-Esteves M. Adeno-associated virus vectors serotyped with AAV8 capsid are more efficient than AAV-1 or -2 serotypes for widespread gene delivery to the neonatal mouse brain. Neuroscience. 2006;138(2):501-10. Epub 2006 Jan 18. PubMed PMID: 16414198. 

2005

Kock N, Allchorne AJ, Sena-Esteves M, Woolf CJ, Breakefield XO. RNAi blocks DYT1 mutant torsinA inclusions in neurons. Neurosci Lett. 2006 Mar 13;395(3):201-5. Epub 2005 Dec 5. PubMed PMID: 16332410.

2004

Steffens S, Tebbets J, Kramm CM, Lindemann D, Flake A, Sena-Esteves M. Transduction of human glial and neuronal tumor cells with different lentivirus vector pseudotypes. J Neurooncol. 2004 Dec;70(3):281-8. PubMed PMID: 15662969. 

Sena-Esteves M, Tebbets JC, Steffens S, Crombleholme T, Flake AW. Optimized large-scale production of high titer lentivirus vector pseudotypes. J Virol Methods. 2004 Dec 15;122(2):131-9. PubMed PMID: 15542136. 

MacKenzie TC, Kobinger GP, Louboutin JP, Radu A, Javazon EH, Sena-Esteves M, Wilson JM, Flake AW. Transduction of satellite cells after prenatal intramuscular administration of lentiviral vectors. J Gene Med. 2005 Jan;7(1):50-8. PubMed PMID: 15515139. 

Bragg DC, Camp SM, Kaufman CA, Wilbur JD, Boston H, Schuback DE, Hanson PI, Sena-Esteves M, Breakefield XO. Perinuclear biogenesis of mutant torsin-A inclusions in cultured cells infected with tetracycline-regulated herpes simplex virus type 1 amplicon vectors. Neuroscience. 2004;125(3):651-61. PubMed PMID: 15099679. 

2003

Hampl JA, Camp SM, Mydlarz WK, Hampl M, Ichikawa T, Chiocca EA, Louis DN, Sena-Esteves M, Breakefield XO. Potentiated gene delivery to tumors using herpes simplex virus/Epstein-Barr virus/RV tribrid amplicon vectors. Hum Gene Ther. 2003 May 1;14(7):611-26. PubMed PMID: 12804144.

2002

Kaye EM, Sena-Esteves M. Gene therapy for the central nervous system in the lysosomal storage disorders. Neurol Clin. 2002 Aug;20(3):879-901. Review. PubMed PMID: 12432834. 

MacKenzie TC, Kobinger GP, Kootstra NA, Radu A, Sena-Esteves M, Bouchard S, Wilson JM, Verma IM, Flake AW. Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes. Mol Ther. 2002 Sep;6(3):349-58. PubMed PMID: 12231171. 

Sena-Esteves M, Hampl JA, Camp SM, Breakefield XO. Generation of stable retrovirus packaging cell lines after transduction with herpes simplex virus hybrid amplicon vectors. J Gene Med. 2002 May-Jun;4(3):229-39. PubMed PMID: 12112640. 

Lim FY, Martin BG, Sena-Esteves M, Radu A, Crombleholme TM. Adeno-associated virus (AAV)-mediated gene transfer in respiratory epithelium and submucosal gland cells in human fetal tracheal organ culture. J Pediatr Surg. 2002 Jul;37(7):1051-7; discussion 1051-7. PubMed PMID: 12077770. 

Hsich G, Sena-Esteves M, Breakefield XO. Critical issues in gene therapy for neurologic disease. Hum Gene Ther. 2002 Mar 20;13(5):579-604. Review. PubMed PMID: 11916483.

2001

Teixeira CA, Sena-Esteves M, Lopes L, Sá Miranda MC, Ribeiro MG. Retrovirus-mediated transfer and expression of beta-hexosaminidase alpha-chain cDNA in human fibroblasts from G(M2)-gangliosidosis B1 variant. Hum Gene Ther. 2001 Sep 20;12(14):1771-83. PubMed PMID: 11560770.

2000

Herrlinger U, Woiciechowski C, Sena-Esteves M, Aboody KS, Jacobs AH, Rainov NG, Snyder EY, Breakefield XO. Neural precursor cells for delivery of replication-conditional HSV-1 vectors to intracerebral gliomas. Mol Ther. 2000 Apr;1(4):347-57. PubMed PMID: 10933953. 

Jacobs A, Dubrovin M, Hewett J, Sena-Esteves M, Tan CW, Slack M, Sadelain M, Breakefield XO, Tjuvajev JG. Functional coexpression of HSV-1 thymidine kinase and green fluorescent protein: implications for noninvasive imaging of transgene expression. Neoplasia. 1999 Jun;1(2):154-61. PubMed PMID: 10933050; PubMed Central PMCID: PMC1508134. 

Herrlinger U, Jacobs A, Quinones A, Woiciechowsky C, Sena-Esteves M, Rainov NG, Fraefel C, Breakefield XO. Helper virus-free herpes simplex virus type 1 amplicon vectors for granulocyte-macrophage colony-stimulating factor-enhanced vaccination therapy for experimental glioma. Hum Gene Ther. 2000 Jul 1;11(10):1429-38. PubMed PMID: 10910140. 

Rainov NG, Koch S, Sena-Esteves M, Berens ME. Characterization of a canine glioma cell line as related to established experimental brain tumor models. J Neuropathol Exp Neurol. 2000 Jul;59(7):607-13. PubMed PMID: 10901232. 

Sena-Esteves M, Saeki Y, Fraefel C, Breakefield XO. HSV-1 amplicon vectors--simplicity and versatility. Mol Ther. 2000 Jul;2(1):9-15. Review. PubMed PMID: 10899823.

Sena-Esteves M, Camp SM, Alroy J, Breakefield XO, Kaye EM. Correction of acid beta-galactosidase deficiency in GM1 gangliosidosis human fibroblasts by retrovirus vector-mediated gene transfer: higher efficiency of release and cross-correction by the murine enzyme. Hum Gene Ther. 2000 Mar 20;11(5):715-27. PubMed PMID: 10757351. 

Klein C, Gurvich N, Sena-Esteves M, Bressman S, Brin MF, Ebersole BJ, Fink S, Forsgren L, Friedman J, Grimes D, Holmgren G, Kyllerman M, Lang AE, de Leon D, Leung J, Prioleau C, Raymond D, Sanner G, Saunders-Pullman R, Vieregge P, Wahlström J, Breakefield XO, Kramer PL, Ozelius LJ, Sealfon SC. Evaluation of the role of the D2 dopamine receptor in myoclonus dystonia. Ann Neurol. 2000 Mar;47(3):369-73. PubMed PMID: 10716258. 

1999

Sena-Esteves M, Saeki Y, Camp SM, Chiocca EA, Breakefield XO. Single-step conversion of cells to retrovirus vector producers with herpes simplex virus-Epstein-Barr virus hybrid amplicons. J Virol. 1999 Dec;73(12):10426-39. PubMed PMID: 10559361; PubMed Central PMCID: PMC113098.

Derby ML, Sena-Esteves M, Breakefield XO, Corey DP. Gene transfer into the mammalian inner ear using HSV-1 and vaccinia virus vectors. Hear Res. 1999 Aug;134(1-2):1-8. PubMed PMID: 10452370.

Klein C, Brin MF, Kramer P, Sena-Esteves M, de Leon D, Doheny D, Bressman S, Fahn S, Breakefield XO, Ozelius LJ. Association of a missense change in the D2 dopamine receptor with myoclonus dystonia. Proc Natl Acad Sci U S A. 1999 Apr 27;96(9):5173-6. PubMed PMID: 10220438; PubMed Central PMCID: PMC21836. 

1998

Rainov NG, Sena-Esteves M, Fraefel C, Dobberstein KU, Chiocca EA, Breakefield XO. A chimeric fusion protein of cytochrome CYP4B1 and green fluorescent protein for detection of pro-drug activating gene delivery and for gene therapy in malignant glioma. Adv Exp Med Biol. 1998;451:393-403. PubMed PMID: 10026902. 

Rainov NG, Dobberstein KU, Sena-Esteves M, Herrlinger U, Kramm CM, Philpot RM, Hilton J, Chiocca EA, Breakefield XO. New prodrug activation gene therapy for cancer using cytochrome P450 4B1 and 2-aminoanthracene/4-ipomeanol. Hum Gene Ther. 1998 Jun 10;9(9):1261-73. PubMed PMID: 9650611. 

1997

Aboody-Guterman KS, Pechan PA, Rainov NG, Sena-Esteves M, Jacobs A, Snyder EY, Wild P, Schraner E, Tobler K, Breakefield XO, Fraefel C. Green fluorescent protein as a reporter for retrovirus and helper virus-free HSV-1 amplicon vector-mediated gene transfer into neural cells in culture and in vivo. Neuroreport. 1997 Dec 1;8(17):3801-8. PubMed PMID: 9427374.

Kramm CM, Chase M, Herrlinger U, Jacobs A, Pechan PA, Rainov NG, Sena-Esteves M, Aghi M, Barnett FH, Chiocca EA, Breakefield XO. Therapeutic efficiency and safety of a second-generation replication-conditional HSV1 vector for brain tumor gene therapy. Hum Gene Ther. 1997 Nov 20;8(17):2057-68. PubMed PMID: 9414254. 

Rage F, Hill DF, Sena-Esteves M, Breakefield XO, Coffey RJ, Costa ME, McCann SM, Ojeda SR. Targeting transforming growth factor alpha expression to discreteloci of the neuroendocrine brain induces female sexual precocity. Proc Natl Acad Sci U S A. 1997 Mar 18;94(6):2735-40. PubMed PMID: 9122266; PubMed Central PMCID: PMC20159. 

1996

Yu JS, Sena-Esteves M, Paulus W, Breakefield XO, Reeves SA. Retroviral delivery and tetracycline-dependent expression of IL-1beta-converting enzyme (ICE) in a rat glioma model provides controlled induction of apoptotic death in tumor cells. Cancer Res. 1996 Dec 1;56(23):5423-7. PubMed PMID: 8968096.

Kramm CM, Rainov NG, Sena-Esteves M, Barnett FH, Chase M, Herrlinger U, Pechan PA, Chiocca EA, Breakefield XO. Long-term survival in a rodent model of disseminated brain tumors by combined intrathecal delivery of herpes vectors and ganciclovir treatment. Hum Gene Ther. 1996 Oct 20;7(16):1989-94. PubMed PMID: 8930659.

Kramm CM, Rainov NG, Sena-Esteves M, Chase M, Pechan PA, Chiocca EA, Breakefield XO. Herpes vector-mediated delivery of marker genes to disseminated central nervous system tumors. Hum Gene Ther. 1996 Feb 10;7(3):291-300. PubMed PMID: 8835217.

1995

Kramm CM, Sena-Esteves M, Barnett FH, Rainov NG, Schuback DE, Yu JS, Pechan PA, Paulus W, Chiocca EA, Breakefield XO. Gene therapy for brain tumors. Brain Pathol. 1995 Oct;5(4):345-81. Review. PubMed PMID: 8974620. 

Fernandes PA, Sena-Esteves M, Moradas-Ferreira P. Characterization of the glyceraldehyde-3-phosphate dehydrogenase gene family from Kluyveromyces marxianus--polymerase chain reaction-single-strand conformation polymorphism as a tool for the study of multigenic families. Yeast. 1995 Jun 30;11(8):725-33. PubMed PMID: 7668042. 

1994

Boviatsis EJ, Park JS, Sena-Esteves M, Kramm CM, Chase M, Efird JT, Wei MX, Breakefield XO, Chiocca EA. Long-term survival of rats harboring brain neoplasms treated with ganciclovir and a herpes simplex virus vector that retains an intact thymidine kinase gene. Cancer Res. 1994 Nov 15;54(22):5745-51. PubMed PMID: 7954393.

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Dr. Miguel Sena-Esteves from the Esteves Lab (Horae Gene Therapy Center) is conducting research and developing therapeutic strategies for rare inherited diseases such as the Tay-Sachs disease

Miguel Seva-Esteves, PhD

 

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