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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. Among authors: bradbury am. 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.
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: bradbury am. 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: bradbury am. ASN Neuro. 2015 Apr 13;7(2):1759091415569908. doi: 10.1177/1759091415569908. Print 2015 Mar-Apr. ASN Neuro. 2015. PMID: 25873306 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. Among authors: bradbury am. 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.
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. Gray-Edwards HL, et al. Among authors: bradbury am. Hum Gene Ther. 2018 Mar;29(3):312-326. doi: 10.1089/hum.2017.163. Epub 2017 Sep 18. Hum Gene Ther. 2018. PMID: 28922945
Canine Models of Inherited Musculoskeletal and Neurodegenerative Diseases.
Story BD, Miller ME, Bradbury AM, Million ED, Duan D, Taghian T, Faissler D, Fernau D, Beecy SJ, Gray-Edwards HL. Story BD, et al. Among authors: bradbury am. Front Vet Sci. 2020 Mar 11;7:80. doi: 10.3389/fvets.2020.00080. eCollection 2020. Front Vet Sci. 2020. PMID: 32219101 Free PMC article. Review.
Krabbe disease successfully treated via monotherapy of intrathecal gene therapy.
Bradbury AM, Bagel JH, Nguyen D, Lykken EA, Pesayco Salvador J, Jiang X, Swain GP, Assenmacher CA, Hendricks IJ, Miyadera K, Hess RS, Ostrager A, ODonnell P, Sands MS, Ory DS, Shelton GD, Bongarzone ER, Gray SJ, Vite CH. Bradbury AM, et al. J Clin Invest. 2020 Sep 1;130(9):4906-4920. doi: 10.1172/JCI133953. J Clin Invest. 2020. PMID: 32773406 Free PMC article.
37 results