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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. Among authors: sands ms. J Clin Invest. 2020 Sep 1;130(9):4906-4920. doi: 10.1172/JCI133953. J Clin Invest. 2020. PMID: 32773406 Free PMC article.
Krabbe disease: New hope for an old disease.
Bradbury AM, Bongarzone ER, Sands MS. Bradbury AM, et al. Among authors: sands ms. 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.
Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease.
Heller GJ, Marshall MS, Issa Y, Marshall JN, Nguyen D, Rue E, Pathmasiri KC, Domowicz MS, van Breemen RB, Tai LM, Cologna SM, Crocker SJ, Givogri MI, Sands MS, Bongarzone ER. Heller GJ, et al. Among authors: sands ms. Mol Ther. 2021 May 5;29(5):1883-1902. doi: 10.1016/j.ymthe.2021.01.026. Epub 2021 Jan 26. Mol Ther. 2021. PMID: 33508430 Free PMC article.
CRISPR-Cas9 Knock-In of T513M and G41S Mutations in the Murine β-Galactosyl-Ceramidase Gene Re-capitulates Early-Onset and Adult-Onset Forms of Krabbe Disease.
Rebiai R, Rue E, Zaldua S, Nguyen D, Scesa G, Jastrzebski M, Foster R, Wang B, Jiang X, Tai L, Brady ST, van Breemen R, Givogri MI, Sands MS, Bongarzone ER. Rebiai R, et al. Among authors: sands ms. Front Mol Neurosci. 2022 May 10;15:896314. doi: 10.3389/fnmol.2022.896314. eCollection 2022. Front Mol Neurosci. 2022. PMID: 35620447 Free PMC article.
152 results