Miguel Sena-Esteves to Genetic Therapy
This is a "connection" page, showing publications Miguel Sena-Esteves has written about Genetic Therapy.
Connection Strength
4.447
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Lahey HG, Webber CJ, Golebiowski D, Izzo CM, Horn E, Taghian T, Rodriguez P, Batista AR, Ellis LE, Hwang M, Martin DR, Gray-Edwards H, Sena-Esteves M. Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice. Mol Ther. 2020 10 07; 28(10):2150-2160.
Score: 0.433
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GuhaSarkar D, Neiswender J, Su Q, Gao G, Sena-Esteves M. Intracranial AAV-IFN-? gene therapy eliminates invasive xenograft glioblastoma and improves survival in orthotopic syngeneic murine model. Mol Oncol. 2017 02; 11(2):180-193.
Score: 0.342
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GuhaSarkar D, Su Q, Gao G, Sena-Esteves M. Systemic AAV9-IFN? gene delivery treats highly invasive glioblastoma. Neuro Oncol. 2016 11; 18(11):1508-1518.
Score: 0.326
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Stoica L, Todeasa SH, Cabrera GT, Salameh JS, ElMallah MK, Mueller C, Brown RH, 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.
Score: 0.322
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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 01; 24(15):4353-64.
Score: 0.304
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Bowers WJ, Breakefield XO, Sena-Esteves M. Genetic therapy for the nervous system. Hum Mol Genet. 2011 Apr 15; 20(R1):R28-41.
Score: 0.228
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Breakefield XO, Sena-Esteves M. Healing genes in the nervous system. Neuron. 2010 Oct 21; 68(2):178-81.
Score: 0.222
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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.
Score: 0.125
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Flotte TR, Cataltepe O, Puri A, Batista AR, Moser R, McKenna-Yasek D, Douthwright C, Gernoux G, Blackwood M, Mueller C, Tai PWL, Jiang X, Bateman S, Spanakis SG, Parzych J, Keeler AM, Abayazeed A, Rohatgi S, Gibson L, Finberg R, Barton BA, Vardar Z, Shazeeb MS, Gounis M, Tifft CJ, Eichler FS, Brown RH, Martin DR, Gray-Edwards HL, Sena-Esteves M. AAV gene therapy for Tay-Sachs disease. Nat Med. 2022 02; 28(2):251-259.
Score: 0.121
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Taghian T, Horn E, Shazeeb MS, Bierfeldt LJ, Tuominen SM, Koehler J, Fernau D, Bertrand S, Frey S, Cataltepe OI, Gounis MJ, Abayazeed AH, Flotte TR, Sena-Esteves M, Gray-Edwards HL. Volume and Infusion Rate Dynamics of Intraparenchymal Central Nervous System Infusion in a Large Animal Model. Hum Gene Ther. 2020 06; 31(11-12):617-625.
Score: 0.108
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Keeler AM, Zieger M, Todeasa SH, McCall AL, Gifford JC, Birsak S, Choudhury SR, Byrne BJ, Sena-Esteves M, ElMallah MK. Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease. Hum Gene Ther. 2019 01; 30(1):57-68.
Score: 0.095
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Volak A, LeRoy SG, Natasan JS, Park DJ, Cheah PS, Maus A, Fitzpatrick Z, Hudry E, Pinkham K, Gandhi S, Hyman BT, Mu D, GuhaSarkar D, Stemmer-Rachamimov AO, Sena-Esteves M, Badr CE, Maguire CA. Virus vector-mediated genetic modification of brain tumor stromal cells after intravenous delivery. J Neurooncol. 2018 Sep; 139(2):293-305.
Score: 0.094
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Gray-Edwards HL, Randle AN, Maitland SA, Benatti HR, Hubbard SM, Canning PF, Vogel MB, Brunson BL, Hwang M, Ellis LE, Bradbury AM, Gentry AS, Taylor AR, Wooldridge AA, Wilhite DR, Winter RL, Whitlock BK, Johnson JA, Holland M, Salibi N, Beyers RJ, Sartin JL, Denney TS, Cox NR, Sena-Esteves M, Martin DR. Adeno-Associated Virus Gene Therapy in a Sheep Model of Tay-Sachs Disease. Hum Gene Ther. 2018 03; 29(3):312-326.
Score: 0.090
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Gray-Edwards HL, Regier DS, Shirley JL, Randle AN, Salibi N, Thomas SE, Latour YL, Johnston J, Golas G, Maguire AS, Taylor AR, Sorjonen DC, McCurdy VJ, Christopherson PW, Bradbury AM, Beyers RJ, Johnson AK, Brunson BL, Cox NR, Baker HJ, Denney TS, Sena-Esteves M, Tifft CJ, Martin DR. Novel Biomarkers of Human GM1 Gangliosidosis Reflect the Clinical Efficacy of Gene Therapy in a Feline Model. Mol Ther. 2017 04 05; 25(4):892-903.
Score: 0.086
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Golebiowski D, van der Bom IMJ, Kwon CS, Miller AD, Petrosky K, Bradbury AM, Maitland S, K?hn AL, Bishop N, Curran E, Silva N, GuhaSarkar D, Westmoreland SV, Martin DR, Gounis MJ, Asaad WF, Sena-Esteves M. Direct Intracranial Injection of AAVrh8 Encoding Monkey ?-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain. Hum Gene Ther. 2017 06; 28(6):510-522.
Score: 0.086
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Bradbury AM, Peterson TA, Gross AL, Wells SZ, McCurdy VJ, Wolfe KG, Dennis JC, Brunson BL, Gray-Edwards H, Randle AN, Johnson AK, Morrison EE, Cox NR, Baker HJ, Sena-Esteves M, Martin DR. AAV-mediated gene delivery attenuates neuroinflammation in feline Sandhoff disease. Neuroscience. 2017 01 06; 340:117-125.
Score: 0.084
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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 08; 24(7):1247-57.
Score: 0.081
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Choudhury SR, Hudry E, Maguire CA, Sena-Esteves M, Breakefield XO, Grandi P. Viral vectors for therapy of neurologic diseases. Neuropharmacology. 2017 Jul 01; 120:63-80.
Score: 0.080
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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.
Score: 0.079
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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.
Score: 0.076
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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.
Score: 0.076
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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 Mar-Apr; 7(2).
Score: 0.076
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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.
Score: 0.073
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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.
Score: 0.073
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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.
Score: 0.071
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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 09; 6(231):231ra48.
Score: 0.071
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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.
Score: 0.065
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Stoica L, Ahmed SS, Gao G, Sena-Esteves M. Gene transfer to the CNS using recombinant adeno-associated virus. Curr Protoc Microbiol. 2013; Chapter 14:14D.5.1-14D.5.18.
Score: 0.065
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Tannous BA, Christensen AP, Pike L, Wurdinger T, Perry KF, Saydam O, Jacobs AH, Garc?a-A?overos J, Weissleder R, Sena-Esteves M, Corey DP, Breakefield XO. Mutant sodium channel for tumor therapy. Mol Ther. 2009 May; 17(5):810-9.
Score: 0.050
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Winkeler A, Sena-Esteves M, Paulis LE, Li H, Waerzeggers Y, R?ckriem 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.
Score: 0.044
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Roy AJ, Leipprandt JR, Patterson JR, Stoll AC, Kemp CJ, Oula ZD, Mola T, Batista AR, Sortwell CE, Sena-Esteves M, Neubig RR. AAV9-Mediated Intrastriatal Delivery of GNAO1 Reduces Hyperlocomotion in Gnao1 Heterozygous R209H Mutant Mice. J Pharmacol Exp Ther. 2024 Jul 18; 390(2):250-259.
Score: 0.036
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Johnson AK, McCurdy VJ, Gray-Edwards HL, Maguire AS, Cochran JN, Gross AL, Skinner HE, Randle AN, Shirley JL, Brunson BL, Bradbury AM, Leroy SG, Hwang M, Rockwell HE, Cox NR, Baker HJ, Seyfried TN, Sena-Esteves M, Martin DR. Life-Limiting Peripheral Organ Dysfunction in Feline Sandhoff Disease Emerges after Effective CNS Gene Therapy. Ann Neurol. 2023 11; 94(5):969-986.
Score: 0.034
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Kell P, Sidhu R, Qian M, Mishra S, Nicoli ER, D'Souza P, Tifft CJ, Gross AL, Gray-Edwards HL, Martin DR, Sena-Esteves M, Dietzen DJ, Singh M, Luo J, Schaffer JE, Ory DS, Jiang X. A pentasaccharide for monitoring pharmacodynamic response to gene therapy in GM1 gangliosidosis. EBioMedicine. 2023 Jun; 92:104627.
Score: 0.033
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McElroy A, Sena-Esteves M, Arjomandnejad M, Keeler AM, Gray-Edwards HL. Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System. Hum Gene Ther. 2022 09; 33(17-18):889-892.
Score: 0.032
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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.
Score: 0.031
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Gross AL, Gray-Edwards HL, Bebout CN, Ta NL, Nielsen K, Brunson BL, Lopez Mercado KR, Osterhoudt DE, Batista AR, Maitland S, Seyfried TN, Sena-Esteves M, Martin DR. Intravenous delivery of adeno-associated viral gene therapy in feline GM1 gangliosidosis. Brain. 2022 04 18; 145(2):655-669.
Score: 0.031
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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.
Score: 0.031
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McCurdy VJ, Johnson AK, Gray-Edwards HL, Randle AN, Bradbury AM, Morrison NE, Hwang M, Baker HJ, Cox NR, Sena-Esteves M, Martin DR. Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease. Gene Ther. 2021 04; 28(3-4):142-154.
Score: 0.027
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McNulty MA, Prevatt PB, Nussbaum ER, Randle AN, Johnson AK, Hudson JA, Gray-Edwards HL, Sena-Esteves M, Martin DR, Carlson CS. Abnormal epiphyseal development in a feline model of Sandhoff disease. J Orthop Res. 2020 12; 38(12):2580-2591.
Score: 0.027
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Taghian T, Marosfoi MG, Puri AS, Cataltepe OI, King RM, Diffie EB, Maguire AS, Martin DR, Fernau D, Batista AR, Kuchel T, Christou C, Perumal R, Chandra S, Gamlin PD, Bertrand SG, Flotte TR, McKenna-Yasek D, Tai PWL, Aronin N, Gounis MJ, Sena-Esteves M, Gray-Edwards HL. A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna. Mol Ther. 2020 02 05; 28(2):411-421.
Score: 0.026
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Mondo E, Moser R, Gao G, Mueller C, Sena-Esteves M, Sapp E, Pfister E, O'Connell D, Takle K, Erger KE, Liu W, Conlon TJ, DiFiglia M, Gounis MJ, Aronin N. Selective Neuronal Uptake and Distribution of AAVrh8, AAV9, and AAVrh10 in Sheep After Intra-Striatal Administration. J Huntingtons Dis. 2018; 7(4):309-319.
Score: 0.023
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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, Cach?n-Gonz?lez 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.
Score: 0.017
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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.
Score: 0.015
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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.
Score: 0.014
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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.
Score: 0.014
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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.
Score: 0.009