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Items: 1 to 50 of 122

1.

Long-Term Therapeutic Efficacy of Intravenous AAV-Mediated Hamartin Replacement in Mouse Model of Tuberous Sclerosis Type 1.

Prabhakar S, Cheah PS, Zhang X, Zinter M, Gianatasio M, Hudry E, Bronson RT, Kwiatkowski DJ, Stemmer-Rachamimov A, Maguire CA, Sena-Esteves M, Tannous BA, Breakefield XO.

Mol Ther Methods Clin Dev. 2019 Aug 16;15:18-26. doi: 10.1016/j.omtm.2019.08.003. eCollection 2019 Dec 13.

2.

Human GLB1 knockout cerebral organoids: A model system for testing AAV9-mediated GLB1 gene therapy for reducing GM1 ganglioside storage in GM1 gangliosidosis.

Latour YL, Yoon R, Thomas SE, Grant C, Li C, Sena-Esteves M, Allende ML, Proia RL, Tifft CJ.

Mol Genet Metab Rep. 2019 Sep 11;21:100513. doi: 10.1016/j.ymgmr.2019.100513. eCollection 2019 Dec.

3.

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.

Alterman JF, Godinho BMDC, Hassler MR, Ferguson CM, Echeverria D, Sapp E, Haraszti RA, Coles AH, Conroy F, Miller R, Roux L, Yan P, Knox EG, Turanov AA, King RM, Gernoux G, Mueller C, Gray-Edwards HL, Moser RP, Bishop NC, Jaber SM, Gounis MJ, Sena-Esteves M, Pai AA, DiFiglia M, Aronin N, Khvorova A.

Nat Biotechnol. 2019 Aug;37(8):884-894. doi: 10.1038/s41587-019-0205-0. Epub 2019 Aug 2.

PMID:
31375812
4.

Preparation of a DNA Standard for Quantitative Polymerase Chain Reaction (qPCR).

Lock M, Korn M, Wilson J, Sena-Esteves M, Gao G.

Cold Spring Harb Protoc. 2019 Aug 1;2019(8):pdb.prot095570. doi: 10.1101/pdb.prot095570.

PMID:
31371468
5.

Measuring the Infectious Titer of Recombinant Adenovirus Using Tissue Culture Infection Dose 50% (TCID50) End-Point Dilution and Quantitative Polymerase Chain Reaction (qPCR).

Lock M, Korn M, Wilson J, Sena-Esteves M, Gao G.

Cold Spring Harb Protoc. 2019 Aug 1;2019(8):pdb.prot095562. doi: 10.1101/pdb.prot095562.

PMID:
31371467
6.

Purification of the Recombinant Adenovirus by Cesium Chloride Gradient Centrifugation.

Su Q, Sena-Esteves M, Gao G.

Cold Spring Harb Protoc. 2019 May 1;2019(5):pdb.prot095547. doi: 10.1101/pdb.prot095547.

PMID:
31043560
7.

Release of the Cloned Recombinant Adenovirus Genome for Rescue and Expansion.

Su Q, Sena-Esteves M, Gao G.

Cold Spring Harb Protoc. 2019 May 1;2019(5):pdb.prot095539. doi: 10.1101/pdb.prot095539.

PMID:
31043559
8.

Construction of Recombinant Adenovirus Genomes by Direct Cloning.

Zhou X, Sena-Esteves M, Gao G.

Cold Spring Harb Protoc. 2019 May 1;2019(5):pdb.prot095521. doi: 10.1101/pdb.prot095521.

PMID:
31043558
9.

Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer.

Wang D, Zhong L, Li M, Li J, Tran K, Ren L, He R, Xie J, Moser RP, Fraser C, Kuchel T, Sena-Esteves M, Flotte TR, Aronin N, Gao G.

Mol Ther Methods Clin Dev. 2018 Oct 4;11:65-72. doi: 10.1016/j.omtm.2018.09.003. eCollection 2018 Dec 14.

10.

Selective Neuronal Uptake and Distribution of AAVrh8, AAV9, and AAVrh10 in Sheep After Intra-Striatal Administration.

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.

J Huntingtons Dis. 2018;7(4):309-319. doi: 10.3233/JHD-180302.

PMID:
30320596
11.

Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease.

Keeler AM, Zieger M, Todeasa SH, McCall AL, Gifford JC, Birsak S, Choudhury SR, Byrne BJ, Sena-Esteves M, ElMallah MK.

Hum Gene Ther. 2019 Jan;30(1):57-68. doi: 10.1089/hum.2018.016. Epub 2018 Jul 25.

PMID:
29901418
12.

Virus vector-mediated genetic modification of brain tumor stromal cells after intravenous delivery.

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.

J Neurooncol. 2018 Sep;139(2):293-305. doi: 10.1007/s11060-018-2889-2. Epub 2018 May 16.

13.

Efficient Gene Silencing in Brain Tumors with Hydrophobically Modified siRNAs.

Osborn MF, Coles AH, Golebiowski D, Echeverria D, Moazami MP, Watts JK, Sena-Esteves M, Khvorova A.

Mol Cancer Ther. 2018 Jun;17(6):1251-1258. doi: 10.1158/1535-7163.MCT-17-1144. Epub 2018 Apr 13.

14.

Monitoring Lentivirus Vector Stocks for Replication-Competent Viruses.

Sena-Esteves M, Gao G.

Cold Spring Harb Protoc. 2018 Apr 2;2018(4). doi: 10.1101/pdb.prot095703.

PMID:
29610364
15.

Titration of Lentivirus Vectors.

Sena-Esteves M, Gao G.

Cold Spring Harb Protoc. 2018 Apr 2;2018(4). doi: 10.1101/pdb.prot095695.

PMID:
29610363
16.

Production of High-Titer Retrovirus and Lentivirus Vectors.

Sena-Esteves M, Gao G.

Cold Spring Harb Protoc. 2018 Apr 2;2018(4). doi: 10.1101/pdb.prot095687.

PMID:
29610362
17.

Patterns of sweetness preference in red wine according to consumer characterisation.

Sena-Esteves MM, Mota M, Malfeito-Ferreira M.

Food Res Int. 2018 Apr;106:38-44. doi: 10.1016/j.foodres.2017.12.043. Epub 2017 Dec 17.

PMID:
29579938
18.

Metallothioneins are neuroprotective agents in lysosomal storage disorders.

Cavalca E, Cesani M, Gifford JC, Sena-Esteves M, Terreni MR, Leoncini G, Peviani M, Biffi A.

Ann Neurol. 2018 Feb;83(2):418-432. doi: 10.1002/ana.25161. Epub 2018 Feb 15.

PMID:
29369397
19.

Cerebral organoids derived from Sandhoff disease-induced pluripotent stem cells exhibit impaired neurodifferentiation.

Allende ML, Cook EK, Larman BC, Nugent A, Brady JM, Golebiowski D, Sena-Esteves M, Tifft CJ, Proia RL.

J Lipid Res. 2018 Mar;59(3):550-563. doi: 10.1194/jlr.M081323. Epub 2018 Jan 22.

20.

Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease.

Pfister EL, DiNardo N, Mondo E, Borel F, Conroy F, Fraser C, Gernoux G, Han X, Hu D, Johnson E, Kennington L, Liu P, Reid SJ, Sapp E, Vodicka P, Kuchel T, Morton AJ, Howland D, Moser R, Sena-Esteves M, Gao G, Mueller C, DiFiglia M, Aronin N.

Hum Gene Ther. 2018 Jun;29(6):663-673. doi: 10.1089/hum.2017.199. Epub 2018 Feb 23.

PMID:
29207890
21.

Epigenetic editing of the Dlg4/PSD95 gene improves cognition in aged and Alzheimer's disease mice.

Bustos FJ, Ampuero E, Jury N, Aguilar R, Falahi F, Toledo J, Ahumada J, Lata J, Cubillos P, Henríquez B, Guerra MV, Stehberg J, Neve RL, Inestrosa NC, Wyneken U, Fuenzalida M, Härtel S, Sena-Esteves M, Varela-Nallar L, Rots MG, Montecino M, van Zundert B.

Brain. 2017 Dec 1;140(12):3252-3268. doi: 10.1093/brain/awx272.

22.

Adeno-Associated Virus Gene Therapy in a Sheep Model of Tay-Sachs Disease.

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.

Hum Gene Ther. 2018 Mar;29(3):312-326. doi: 10.1089/hum.2017.163. Epub 2017 Sep 18.

PMID:
28922945
23.

Lipidomic Evaluation of Feline Neurologic Disease after AAV Gene Therapy.

Gray-Edwards HL, Jiang X, Randle AN, Taylor AR, Voss TL, Johnson AK, McCurdy VJ, Sena-Esteves M, Ory DS, Martin DR.

Mol Ther Methods Clin Dev. 2017 Jul 26;6:135-142. doi: 10.1016/j.omtm.2017.07.005. eCollection 2017 Sep 15.

24.

Restrictive Lung Disease in the Cu/Zn Superoxide-Dismutase 1 G93A Amyotrophic Lateral Sclerosis Mouse Model.

Stoica L, Keeler AM, Xiong L, Kalfopoulos M, Desrochers K, Brown RH Jr, Sena-Esteves M, Flotte TR, ElMallah MK.

Am J Respir Cell Mol Biol. 2017 Mar;56(3):405-408. doi: 10.1165/rcmb.2016-0258LE. No abstract available.

25.

Novel Biomarkers of Human GM1 Gangliosidosis Reflect the Clinical Efficacy of Gene Therapy in a Feline Model.

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.

Mol Ther. 2017 Apr 5;25(4):892-903. doi: 10.1016/j.ymthe.2017.01.009. Epub 2017 Feb 22.

26.

Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain.

Golebiowski D, van der Bom IMJ, Kwon CS, Miller AD, Petrosky K, Bradbury AM, Maitland S, Kühn AL, Bishop N, Curran E, Silva N, GuhaSarkar D, Westmoreland SV, Martin DR, Gounis MJ, Asaad WF, Sena-Esteves M.

Hum Gene Ther. 2017 Jun;28(6):510-522. doi: 10.1089/hum.2016.109. Epub 2017 Jan 26.

27.

Intracranial AAV-IFN-β gene therapy eliminates invasive xenograft glioblastoma and improves survival in orthotopic syngeneic murine model.

GuhaSarkar D, Neiswender J, Su Q, Gao G, Sena-Esteves M.

Mol Oncol. 2017 Feb;11(2):180-193. doi: 10.1002/1878-0261.12020. Epub 2017 Jan 18.

28.

AAV-mediated gene delivery attenuates neuroinflammation in feline Sandhoff disease.

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.

Neuroscience. 2017 Jan 6;340:117-125. doi: 10.1016/j.neuroscience.2016.10.047. Epub 2016 Oct 26.

29.

Cellular Analysis of Silencing the Huntington's Disease Gene Using AAV9 Mediated Delivery of Artificial Micro RNA into the Striatum of Q140/Q140 Mice.

Keeler AM, Sapp E, Chase K, Sottosanti E, Danielson E, Pfister E, Stoica L, DiFiglia M, Aronin N, Sena-Esteves M.

J Huntingtons Dis. 2016 Oct 1;5(3):239-248.

PMID:
27689620
30.

Autophagy Activation by Transcription Factor EB (TFEB) in Striatum of HDQ175/Q7 Mice.

Vodicka P, Chase K, Iuliano M, Tousley A, Valentine DT, Sapp E, Kegel-Gleason KB, Sena-Esteves M, Aronin N, DiFiglia M.

J Huntingtons Dis. 2016 Oct 1;5(3):249-260.

31.

Adeno Associated Viral Vector Delivered RNAi for Gene Therapy of SOD1 Amyotrophic Lateral Sclerosis.

Stoica L, Sena-Esteves M.

Front Mol Neurosci. 2016 Aug 2;9:56. doi: 10.3389/fnmol.2016.00056. eCollection 2016. Review.

32.

Effects of Exogenous NUB1 Expression in the Striatum of HDQ175/Q7 Mice.

Vodicka P, Chase K, Iuliano M, Valentine DT, Sapp E, Lu B, Kegel-Gleason KB, Sena-Esteves M, Aronin N, DiFiglia M.

J Huntingtons Dis. 2016 Jun 13;5(2):163-74. doi: 10.3233/JHD-160195.

PMID:
27314618
33.

Systemic AAV9-IFNβ gene delivery treats highly invasive glioblastoma.

GuhaSarkar D, Su Q, Gao G, Sena-Esteves M.

Neuro Oncol. 2016 Nov;18(11):1508-1518. Epub 2016 May 18.

34.

In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy.

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.

Mol Ther. 2016 Aug;24(7):1247-57. doi: 10.1038/mt.2016.84. Epub 2016 Apr 27.

35.

Viral vectors for therapy of neurologic diseases.

Choudhury SR, Hudry E, Maguire CA, Sena-Esteves M, Breakefield XO, Grandi P.

Neuropharmacology. 2017 Jul 1;120:63-80. doi: 10.1016/j.neuropharm.2016.02.013. Epub 2016 Feb 21. Review.

36.

Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model.

Stoica L, Todeasa SH, Cabrera GT, Salameh JS, ElMallah MK, Mueller C, Brown RH Jr, Sena-Esteves M.

Ann Neurol. 2016 Apr;79(4):687-700. doi: 10.1002/ana.24618. Epub 2016 Mar 11.

37.

Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector.

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.

Mol Ther. 2016 Apr;24(4):726-35. doi: 10.1038/mt.2015.231. Epub 2015 Dec 28.

38.

Survival benefit and phenotypic improvement by hamartin gene therapy in a tuberous sclerosis mouse brain model.

Prabhakar S, Zhang X, Goto J, Han S, Lai C, Bronson R, Sena-Esteves M, Ramesh V, Stemmer-Rachamimov A, Kwiatkowski DJ, Breakefield XO.

Neurobiol Dis. 2015 Oct;82:22-31. doi: 10.1016/j.nbd.2015.04.018. Epub 2015 May 24.

39.

Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy.

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.

Mol Genet Metab. 2015 Sep-Oct;116(1-2):80-7. doi: 10.1016/j.ymgme.2015.05.003. Epub 2015 May 8.

PMID:
25971245
40.

Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan.

Weismann CM, Ferreira J, Keeler AM, Su Q, Qui L, Shaffer SA, Xu Z, Gao G, Sena-Esteves M.

Hum Mol Genet. 2015 Aug 1;24(15):4353-64. doi: 10.1093/hmg/ddv168. Epub 2015 May 10.

41.

AAV-mediated gene delivery in a feline model of Sandhoff disease corrects lysosomal storage in the central nervous system.

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.

ASN Neuro. 2015 Apr 13;7(2). pii: 1759091415569908. doi: 10.1177/1759091415569908. Print 2015 Mar-Apr.

42.

Bis(monoacylglycero)phosphate: a secondary storage lipid in the gangliosidoses.

Akgoc Z, Sena-Esteves M, Martin DR, Han X, d'Azzo A, Seyfried TN.

J Lipid Res. 2015 May;56(5):1006-13. doi: 10.1194/jlr.M057851. Epub 2015 Mar 20.

43.

Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease.

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.

Gene Ther. 2015 Feb;22(2):181-9. doi: 10.1038/gt.2014.108. Epub 2014 Dec 4.

PMID:
25474439
44.

Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff disease.

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.

Exp Neurol. 2015 Jan;263:102-12. doi: 10.1016/j.expneurol.2014.09.020. Epub 2014 Oct 5.

45.

Gene therapy approach to FAP: in vivo influence of T119M in TTR deposition in a transgenic V30M mouse model.

Batista AR, Gianni D, Ventosa M, Coelho AV, Almeida MR, Sena-Esteves M, Saraiva MJ.

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.

PMID:
25273354
46.

Gene therapy for the nervous system: challenges and new strategies.

Maguire CA, Ramirez SH, Merkel SF, Sena-Esteves M, Breakefield XO.

Neurotherapeutics. 2014 Oct;11(4):817-39. doi: 10.1007/s13311-014-0299-5. Review.

47.

Ganglioside storage diseases: on the road to management.

Seyfried TN, Rockwell HE, Heinecke KA, Martin DR, Sena-Esteves M.

Adv Neurobiol. 2014;9:485-99. doi: 10.1007/978-1-4939-1154-7_22.

PMID:
25151393
48.

Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.

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.

Hum Gene Ther. 2014 Jun;25(6):488-97. doi: 10.1089/hum.2014.045. Review.

49.

Sustained normalization of neurological disease after intracranial gene therapy in a feline model.

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.

Sci Transl Med. 2014 Apr 9;6(231):231ra48. doi: 10.1126/scitranslmed.3007733.

50.

Hypothalamic dysfunction of the thrombospondin receptor α2δ-1 underlies the overeating and obesity triggered by brain-derived neurotrophic factor deficiency.

Cordeira JW, Felsted JA, Teillon S, Daftary S, Panessiti M, Wirth J, Sena-Esteves M, Rios M.

J Neurosci. 2014 Jan 8;34(2):554-65. doi: 10.1523/JNEUROSCI.1572-13.2014.

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