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2021 | 1 |
2022 | 1 |
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2024 | 1 |
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Krabbe disease: New hope for an old disease.
Neurosci Lett. 2021 May 1;752:135841. doi: 10.1016/j.neulet.2021.135841. Epub 2021 Mar 22.
Neurosci Lett. 2021.
PMID: 33766733
Free PMC article.
Review.
Preclinical studies in Krabbe disease: A model for the investigation of novel combination therapies for lysosomal storage diseases.
Heller G, Bradbury AM, Sands MS, Bongarzone ER.
Heller G, et al.
Mol Ther. 2023 Jan 4;31(1):7-23. doi: 10.1016/j.ymthe.2022.09.017. Epub 2022 Oct 4.
Mol Ther. 2023.
PMID: 36196048
Free PMC article.
Review.
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Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy.
Bradbury AM, Bagel J, Swain G, Miyadera K, Pesayco JP, Assenmacher CA, Brisson B, Hendricks I, Wang XH, Herbst Z, Pyne N, Odonnell P, Shelton GD, Gelb M, Hackett N, Szabolcs P, Vite CH, Escolar M.
Bradbury AM, et al.
Mol Ther. 2024 Jan 3;32(1):44-58. doi: 10.1016/j.ymthe.2023.11.014. Epub 2023 Nov 11.
Mol Ther. 2024.
PMID: 37952085
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