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Your search keyword '"Escandon, Rafael"' showing total 15 results
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15 results on '"Escandon, Rafael"'

3. Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies – Developing Potential Treatments for the Entire Spectrum of Disease

Author

McDonald, Craig, primary, Camino, Eric, additional, Escandon, Rafael, additional, Finkel, Richard S., additional, Fischer, Ryan, additional, Flanigan, Kevin, additional, Furlong, Pat, additional, Juhasz, Rose, additional, Martin, Ann S., additional, Villa, Chet, additional, and Sweeney, H. Lee, additional

Published
2024
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10. Design of a Phase 1/2 Open-Label, Dose-Escalation Study of the Safety and Efficacy of Gene Therapy in Adults With Classic Congenital Adrenal Hyperplasia (CAH) Due to 21-hydroxylase Deficiency Through Administration of an Adeno-Associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene

Author

Merke, Deborah P, primary, Auchus, Richard Joseph, additional, Sarafoglou, Kyriakie, additional, Geffner, Mitchell E, additional, Kim, Mimi S, additional, Escandon, Rafael D, additional, Bharucha, Kamal N, additional, Shaywitz, Adam J, additional, Eclov, Rachel, additional, Beard, Clayton W, additional, Fur, Sophie Le, additional, and Bougneres, Pierre F, additional

Published
2021
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15. The novel kinesin spindle protein (KSP) inhibitor SB-743921 exhibits marked activity in in vivo and in vitro models of aggressive large B-cell lymphoma.

Author

Bongero, Danielle, Paoluzzi, Luca, Marchi, Enrica, Zullo, Kelly M., Neisa, Roberto, Mao, Yinghui, Escandon, Rafael, Wood, Ken, and O'Connor, Owen A.

Subjects
DIFFUSE large B-cell lymphomas, KINESIN, CELL-mediated cytotoxicity, GERMINAL centers, CELL lines, CELL proliferation, APOPTOSIS, XENOGRAFTS
Abstract

The kinesin spindle protein (KSP) is a mitotic protein essential for cell cycle control and motility. SB-743921 (hereafter SB-921) is an inhibitor that selectively targets the ATP-binding domain of the KSP. The preclinical activity of SB-921 was evaluated in models of diffuse large B-cell lymphoma (DLBCL). The cytotoxicity of SB-921 was evaluated in a series of germinal center (GC-DLBCL) and post-germinal center (ABC-DLBCL) DLBCL cell lines and a murine lymphoma xenograft model. GC-DLBCL lines generally demonstrated greater sensitivity to SB-921. IC50values ranged between 1 nM and 900 nM for GC-DLBCL compared to 1 nM to 10 μM for ABC lines. SB-921 demonstrated marked activity in a xenograft model of Ly-1 (GC-DLBCL). While SB-921 was relatively more active in GC derived cell lines, ABC-derived lines still underwent apoptosis at higher concentrations. These results demonstrate that SB-921 inhibits proliferation and induces apoptosis in both GC-DLBCL and ABC-DLBCL. [ABSTRACT FROM AUTHOR]

Published
2015
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