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Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe's Disease after Global Gene Therapy.
Marshall MS, Issa Y, Jakubauskas B, Stoskute M, Elackattu V, Marshall JN, Bogue W, Nguyen D, Hauck Z, Rue E, Karumuthil-Melethil S, Zaric V, Bosland M, van Breemen RB, Givogri MI, Gray SJ, Crocker SJ, Bongarzone ER. Marshall MS, et al. Among authors: gray sj. Mol Ther. 2018 Mar 7;26(3):874-889. doi: 10.1016/j.ymthe.2018.01.009. Epub 2018 Jan 17. Mol Ther. 2018. PMID: 29433937 Free PMC article.
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: gray sj. J Clin Invest. 2020 Sep 1;130(9):4906-4920. doi: 10.1172/JCI133953. J Clin Invest. 2020. PMID: 32773406 Free PMC article.
316 results