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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: bongarzone er. 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.
The Pathogenic Sphingolipid Psychosine is Secreted in Extracellular Vesicles in the Brain of a Mouse Model of Krabbe Disease.
Reiter CR, Rebiai R, Kwak A, Marshall J, Wozniak D, Scesa G, Nguyen D, Rue E, Pathmasiri C, Pijewski R, van Breemen R, Cologna S, Crocker SJ, Givogri MI, Bongarzone ER. Reiter CR, et al. Among authors: bongarzone er. ASN Neuro. 2022 Jan-Dec;14:17590914221087817. doi: 10.1177/17590914221087817. ASN Neuro. 2022. PMID: 35300522 Free PMC article.
Krabbe disease: New hope for an old disease.
Bradbury AM, Bongarzone ER, Sands MS. Bradbury AM, et al. Among authors: bongarzone er. 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.
Synaptic Function and Dysfunction in Lysosomal Storage Diseases.
Rebiai R, Givogri MI, Gowrishankar S, Cologna SM, Alford ST, Bongarzone ER. Rebiai R, et al. Among authors: bongarzone er. Front Cell Neurosci. 2021 Mar 4;15:619777. doi: 10.3389/fncel.2021.619777. eCollection 2021. Front Cell Neurosci. 2021. PMID: 33746713 Free PMC article.
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: bongarzone er. 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.
94 results