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455 results

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Page 1
Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy.
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. Bradbury AM, et al. Among authors: martin dr. Mol Ther. 2013 Jul;21(7):1306-15. doi: 10.1038/mt.2013.86. Epub 2013 May 21. Mol Ther. 2013. PMID: 23689599 Free PMC article.
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. McCurdy VJ, et al. Among authors: martin dr. Sci Transl Med. 2014 Apr 9;6(231):231ra48. doi: 10.1126/scitranslmed.3007733. Sci Transl Med. 2014. PMID: 24718858 Free PMC article.
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. Bradbury AM, et al. Among authors: martin dr. Exp Neurol. 2015 Jan;263:102-12. doi: 10.1016/j.expneurol.2014.09.020. Epub 2014 Oct 5. Exp Neurol. 2015. PMID: 25284324 Free PMC article.
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. McCurdy VJ, et al. Among authors: martin dr. Gene Ther. 2015 Feb;22(2):181-9. doi: 10.1038/gt.2014.108. Epub 2014 Dec 4. Gene Ther. 2015. PMID: 25474439
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. Rockwell HE, et al. Among authors: martin dr. ASN Neuro. 2015 Apr 13;7(2):1759091415569908. doi: 10.1177/1759091415569908. Print 2015 Mar-Apr. ASN Neuro. 2015. PMID: 25873306 Free PMC article.
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. Gray-Edwards HL, et al. Among authors: 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. Mol Genet Metab. 2015. PMID: 25971245
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. Choudhury SR, et al. Among authors: martin dr. Mol Ther. 2016 Apr;24(4):726-35. doi: 10.1038/mt.2015.231. Epub 2015 Dec 28. Mol Ther. 2016. PMID: 26708003 Free PMC article.
455 results