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Ganglioside storage diseases: on the road to management.
Seyfried TN, Rockwell HE, Heinecke KA, Martin DR, Sena-Esteves M. Seyfried TN, et al. Among authors: martin dr. Adv Neurobiol. 2014;9:485-99. doi: 10.1007/978-1-4939-1154-7_22. Adv Neurobiol. 2014. PMID: 25151393
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.
Emerging therapies for neuropathic lysosomal storage disorders.
Kelly JM, Bradbury A, Martin DR, Byrne ME. Kelly JM, et al. Among authors: martin dr. Prog Neurobiol. 2017 May;152:166-180. doi: 10.1016/j.pneurobio.2016.10.002. Epub 2016 Oct 8. Prog Neurobiol. 2017. PMID: 27725193 Review.
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. Among authors: martin dr. 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. Among authors: martin dr. 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.
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.
Evaluation of N-nonyl-deoxygalactonojirimycin as a pharmacological chaperone for human GM1 gangliosidosis leads to identification of a feline model suitable for testing enzyme enhancement therapy.
Rigat BA, Tropak MB, Buttner J, Crushell E, Benedict D, Callahan JW, Martin DR, Mahuran DJ. Rigat BA, et al. Among authors: martin dr. Mol Genet Metab. 2012 Sep;107(1-2):203-12. doi: 10.1016/j.ymgme.2012.06.007. Epub 2012 Jun 19. Mol Genet Metab. 2012. PMID: 22784478 Free PMC article.
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