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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: marshall jn. 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.
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: marshall jn. 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.