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Page 1
Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders.
Davidson BL, Gao G, Berry-Kravis E, Bradbury AM, Bönnemann C, Buxbaum JD, Corcoran GR, Gray SJ, Gray-Edwards H, Kleiman RJ, Shaywitz AJ, Wang D, Zoghbi HY, Flotte TR, Tauscher-Wisniewski S, Tifft CJ, Sahin M; Gene Therapy Workshop Faculty. Davidson BL, et al. Mol Ther. 2022 Jul 6;30(7):2416-2428. doi: 10.1016/j.ymthe.2022.05.014. Epub 2022 May 17. Mol Ther. 2022. PMID: 35585789 Free PMC article. Review.
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. 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.
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. 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.
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. 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. 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
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. Choudhury SR, et al. Mol Ther. 2016 Aug;24(7):1247-57. doi: 10.1038/mt.2016.84. Epub 2016 Apr 27. Mol Ther. 2016. PMID: 27117222 Free PMC article.
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. Bradbury AM, et al. Neuroscience. 2017 Jan 6;340:117-125. doi: 10.1016/j.neuroscience.2016.10.047. Epub 2016 Oct 26. Neuroscience. 2017. PMID: 27793778 Free PMC article.
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. Gray-Edwards HL, et al. Mol Ther. 2017 Apr 5;25(4):892-903. doi: 10.1016/j.ymthe.2017.01.009. Epub 2017 Feb 22. Mol Ther. 2017. PMID: 28236574 Free PMC article.
50 results