Journal Articles
1. Bradbury AM, Morrison NE, Hwang M, Cox NR, Baker HJ and Martin DR. Neurodegenerative lysosomal storage disease in European Burmese cats with hexosaminidase beta-subunit deficiency. Mol Genet Metab 97(1):53-9, 2009. PMID 19231264
2. Gray-Edwards HL, Salibi N, Josephson EM, Hudson JA, Cox NR, Randle AN, McCurdy VJ, Bradbury AM, Wilson DU, Beyers RJ, Denney TS and Martin DR. High resolution MRI anatomy of the cat brain at 3Tesla. J Neurosci Methods 227:10-17, 2014. PMID 24525327
3. Seyfried TN, Rockwell HE, Heinicke KA, Martin DR, Sena-Esteves M. Ganglioside Storage Diseases: On the Road to Management, in Advances in Neurobiology: Glycobiology of the Nervous System (Shengrund, ed.) 9:485-99, 2014. PMID 25151393
4. Rockwell H, McCurdy VJ, Eaton S, Wilson DU, Johnson AK, Randle AN, Bradbury AM, Gray-Edwards HL, Baker HJ, Hudson JA, Cox NR, Sena-Esteves M and Martin DR. AAV-mediated gene delivery in a feline model of Sandhoff disease corrects lysosomal storage in the central nervous system. ASN Neuro 7(2): pii: 1759091415569908, 2015. PMID 25873306
5. McCurdy VJ, Rockwell HE, Arthur JA, Bradbury AM, Johnson AK, Randle AN, Brunson BL, Hwang M, Gray-Edwards HL, Morrison NE, Johnson JA, Cox NR, Baker HJ, Seyfried TN, Sena-Esteves M and Martin DR. Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease. Gene Ther 22(2):181-9, 2015. PMID 25474439
6. Bradbury AM, Heather Gray-Edwards H, McCurdy VJ, Shirley JL, Colaco AN, Randle AN, Christopherson PW, Bird AC, De Pompa NL, Sorjonen DC, Brunson BL, Jeyakumar M, Platt FM, Cox NR, Baker HJ, Sena-Esteves and Martin DR. Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff disease. Exp Neurol 263:102-12, 2015. PMID 25284324
7. 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, 20:pii: jlr.M057851, 2015. PMID 25795792
8. Gray-Edwards HL, Brunson BL, Holland M, Hespel AM, Bradbury AM, Salibi N, Denney TS, Beyers RJ, Johnson AK, Voyles ML, Montgomery RD, Wilson DU, Hudson JA, Cox NR, Baker HJ, Sena-Esteves M and Martin DR. Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy. Mol Genet Metab, 16(1-2):80-7, 2015. PMID 25971245
Book Chapter
1.Golebiowski D, Bradbury AM, Kwon CS, van der Bom IMJ, Stoica L, Johnson AK, Wilson DU, Gray-Edwards HL, Hudson JA, Johnson JA, Randle AN, Whitlock BK, Sartin JL, Kühn AL, Gounis M, Asaad W, Martin DR and Sena-Esteves M. AAV gene therapy strategies for lysosomal storage disorders with central nervous system involvement, in Neuromethods: Gene Delivery and Therapy for Neurological Disorders (Bo X, Verhaagen J, eds.) 98:265-295, 2015.
Golebiowski D, Bradbury AM, Kwon CS, van der Bom IMJ, Stoica L, Johnson AK, Wilson DU, Gray-Edwards HL, Hudson JA, Johnson JA, Randle AN, Whitlock BK, Sartin JL, Kühn AL, Gounis M, Asaad W, Martin DR and Sena-Esteves M. AAV gene therapy strategies for lysosomal storage disorders with central nervous system involvement, in Neuromethods: Gene Delivery and Therapy for Neurological Disorders (Bo X, Verhaagen J, eds.) 98:265-295, 2015.Roos, J.C., Lachmann, R.H., Carpenter, R.H., Cox, T.M. (2011) Latency vs. Saccadic Parameters in Lysosomal Trials. Opthalmology 118(4): 794.
Sargeant, T.J., Wang, S., Bradley, J., Smith, N.J., Raha, A.A., McNair, R., Ziegler, R.J., Cheng, S.H., Cox, T.M., Cachón-González, M.B. (2011) Adeno-Associated Virus-Mediated Expression of β-Hexosaminidase Prevents Neuronal Loss in the Sandhoff Mouse Brain. Hum Mol Genet. 20(22):4371-80. Epub Aug 18.
Smith, N.J., Winstone, A.M., Stellitano, L., Cox, T.M., Verity, C.M. (2012) GM2 Gangliosidosis in a UK Study of Children with Progressive Neurodegeneration: 73 Cases Reviewed. Dev Med Child Neurol. 54(2):176-82. Epub 2011 Nov 24
Cachón-González, M.B., Wang, S.Z., McNair, R., Bradley, J., Lunn, D., Ziegler, R., Cheng, S.H., Cox, T.M. (2012) Gene Transfer Corrects Acute GM2 Gangliosidosis--Potential Therapeutic Contribution of Perivascular Enzyme Flow. Mol Ther. 20(8):1489-500. Epub Mar 27
Bley, A.E., Giannikopoulos, O.A., Hayden, D., Kubilus, K., Tifft, C.J., Eichler, F.S. (2011) Natural History of Infantile G(M2) Gangliosidosis. Pediatrics 128(5):e1233-41
Sargeant, T.J., Drage, D.J., Wang, S., Apostolakis, A.A., Cox, T.M., Cachón-González, M.B. (2012) Characterization of Inducible Models of Tay-Sachs and Related Disease. PLoS Genet. Sep;8(9):e1002943. Epub 2012 Sep 20.
Cox, T.M., Cachón-González, M.B. (2012) The Cellular Pathology of Lysosomal Diseases. J Pathol 226(2):241-54.
Bradbury, A.M., Brunson, B.L., McCurdy, V.J., Gray-Edwards, H., Seyfried, T.N., Cox, N.R., Baker, H.J., Sena-Esteves, M., Martin, D.R. (2013) Dramatic Therapeutic Response of Intracranial AAV Gene Therapy in Feline GM2 Gangliosidosis. Gordon Research Seminar and Conference: Lysosomal Diseases, Lucca (Barga), Italy: April 13-19, 2013
Bradbury, A.M., McCurdy, V.J., Johnson, A.K., Gray-Edwards, H., Brunson, B.L, Randle, A.N, Cox, N.R., Sena-Esteves, M., Martin, D.R. Intracranial AAV Gene Therapy Extends the Life Span of GM2 Gangliosidosis Cats >Four-Fold with No Clinical Evidence of Vector Toxicity. (2013) Sixteenth Annual Meeting of the American Society of Gene and Cell Therapy, Salt Lake City, UT: May 15-18, 2013.
McCurdy, V.J., Gray-Edwards, H., Randle, A.N, Bradbury, A.M., Johnson, A.K., Beadlescomb, P.M., Morrison, N.E., Hwang, M., Sena-Esteves, M., Martin, D.R. (2013) Dramatic Phenotypic Improvement after Adeno-Associated Virus Gene Therapy in a Feline Model of Sandhoff Disease. Sixteenth Annual Meeting of the American Society of Gene and Cell Therapy, Salt Lake City, UT: May 15-18, 2013.
Gray-Edwards, H. L., Hubbard, S. M., Randle, Hwang, M., Bradbury, A. M., McCurdy, V. J., Wilson, D. U., Hudson, J. A., Whitlock, B.K., Sartin, J. L., Salibi, N., Beyers, R. J., Denney, T. S., Johnson, J. A., Riddell, K. P., Cox, N. R., Sena-Estevez, M., Martin, D. R. (2013) Amelioration of Neurologic Disease after AAV-Gene Therapy in a Sheep Model of Tay-Sachs Disease. Sixteenth Annual Meeting of the American Society of Gene and Cell Therapy, Salt Lake City, UT: May 15-18, 2013.
McCurdy, V.J., Randle, A.N, Gray-Edwards, H., Bradbury, A.M., Johnson, A.K., Beadlescomb, P.M., Sena-Esteves M., Martin D.R. (2013) Significant Therapeutic Benefit after Post-Symptomatic Gene Therapy in a Feline Model of GM2 Gangliosidosis. Sixteenth Annual Meeting of the American Society of Gene and Cell Therapy, Salt Lake City, UT: May 15-18, 2013.
Gray-Edwards, H., Bradbury, A.M., McCurdy, V.J., Wilson, D. Brunson, B., Holland, M., Salibi, N., Beyers, R, Denny, T., Randle, A.N., Johnson, A.K., Cox, N.R., Sena-Esteves, M., Martin, D.R. (2013) Marked Attenuation of Neurologic Disease and Emergence of Peripheral Disease After Brain-Directed Gene Therapy in a Feline Model of Sandhoff Disease. (2013) Gordon Research Seminar and Conference: Lysosomal Diseases April 13-19, 2013
Weismann, C.M., Ferreirra, J., Maitland, S., Su, Q., Guangping, G., Sena-Esteves, M. (2013) Intravenous Administration of AAV9-mβgal in a GM1 Gangliosidosis Mouse Model Expresses Enzyme in the CNS and Delays Disease Onset with Gender Differences. ASGCT Meeting, May 15-18, 2013
Golebiowski, D., van der Bom, I.M.J., Su-Kown, C., Maitland, S., Kühn, A.L., Bishop, N., Curren, E., Silva, N. Miller, A., Westmoreland, S., Gounis, M., Martin, D.R., Asaad, W., Sena-Esteves, M. (2013) Safety Study of Intracranial AAVrh8-Mediated Gene Transfer of beta-Hexosaminidase in Non-Human Primates. ASGCT Meeting, May 15-18, 2013
Erger, K.E., Mandel, R.J., Castleman, W.L., Campbell-Thompson, M., Abernathy, C., Getz, M.P., Cossette, T.L., Schneider, L., Brown, R., Sena-Esteves, M., Conlon, T.J. (2013) Biodistribution and Toxicology Studies of AAV2/rh8-CBA-β-Hexosaminidase after Intracranial Delivery in C57/Bl6 Mice. ASGCT Meeting, May 15-18, 2013
Rena C. Baek, Douglas R. Martin, Nancy R. Cox, Thomas N. Seyfried
Published Articles and Publications
& Publications
