Below are the most recent publications written about "Gangliosidosis, GM1" by people in Profiles.
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Kolstad J, Zoppo C, Johnston JM, D'Souza P, K?hn AL, Vardar Z, Peker A, Hader A, Celik H, Lewis CJ, Lindsay C, Rentiya ZS, Lebel C, Vedantham S, Vachha B, Gray-Edwards HL, Acosta MT, Tifft CJ, Shazeeb MS. Natural history progression of MRI brain volumetrics in type II late-infantile and juvenile GM1 gangliosidosis patients. Mol Genet Metab. 2025 Mar; 144(3):109025.
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Kell P, Sidhu R, Qian M, Mishra S, Nicoli ER, D'Souza P, Tifft CJ, Gross AL, Gray-Edwards HL, Martin DR, Sena-Esteves M, Dietzen DJ, Singh M, Luo J, Schaffer JE, Ory DS, Jiang X. A pentasaccharide for monitoring pharmacodynamic response to gene therapy in GM1 gangliosidosis. EBioMedicine. 2023 Jun; 92:104627.
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Nicoli ER, Huebecker M, Han ST, Garcia K, Munasinghe J, Lizak M, Latour Y, Yoon R, Glase B, Tyrlik M, Peiravi M, Springer D, Baker EH, Priestman D, Sidhu R, Kell P, Jiang X, Kolstad J, Kuhn AL, Shazeeb MS, Acosta MT, Proia RL, Platt FM, Tifft CJ. Glb1 knockout mouse model shares natural history with type II GM1 gangliosidosis patients. Mol Genet Metab. 2023 02; 138(2):107508.
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Gross AL, Gray-Edwards HL, Bebout CN, Ta NL, Nielsen K, Brunson BL, Lopez Mercado KR, Osterhoudt DE, Batista AR, Maitland S, Seyfried TN, Sena-Esteves M, Martin DR. Intravenous delivery of adeno-associated viral gene therapy in feline GM1 gangliosidosis. Brain. 2022 04 18; 145(2):655-669.
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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. Novel Biomarkers of Human GM1 Gangliosidosis Reflect the Clinical Efficacy of Gene Therapy in a Feline Model. Mol Ther. 2017 04 05; 25(4):892-903.
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Weismann CM, Ferreira J, Keeler AM, Su Q, Qui L, Shaffer SA, Xu Z, Gao G, Sena-Esteves M. Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan. Hum Mol Genet. 2015 Aug 01; 24(15):4353-64.
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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. 2015 May; 56(5):1006-13.
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Susuki K, Yuki N, Schafer DP, Hirata K, Zhang G, Funakoshi K, Rasband MN. Dysfunction of nodes of Ranvier: a mechanism for anti-ganglioside antibody-mediated neuropathies. Exp Neurol. 2012 Jan; 233(1):534-42.
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Baek RC, Broekman ML, Leroy SG, Tierney LA, Sandberg MA, d'Azzo A, Seyfried TN, Sena-Esteves M. AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival. PLoS One. 2010 Oct 18; 5(10):e13468.
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Kaye EM, Alroy J, Raghavan SS, Schwarting GA, Adelman LS, Runge V, Gelblum D, Thalhammer JG, Zuniga G. Dysmyelinogenesis in animal model of GM1 gangliosidosis. Pediatr Neurol. 1992 Jul-Aug; 8(4):255-61.