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Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations.
Yoon JK, Schindler JW, Loperfido M, Baricordi C, DeAndrade MP, Jacobs ME, Treleaven C, Plasschaert RN, Yan A, Barese CN, Dogan Y, Chen VP, Fiorini C, Hull F, Barbarossa L, Unnisa Z, Ivanov D, Kutner RH, Guda S, Oborski C, Maiwald T, Michaud V, Rothe M, Schambach A, Pfeifer R, Mason C, Biasco L, van Til NP. Yoon JK, et al. Among authors: guda s. Mol Ther. 2024 Nov 6;32(11):3847-3864. doi: 10.1016/j.ymthe.2024.09.024. Epub 2024 Sep 17. Mol Ther. 2024. PMID: 39295144
Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease.
Dogan Y, Barese CN, Schindler JW, Yoon JK, Unnisa Z, Guda S, Jacobs ME, Oborski C, Maiwald T, Clarke DL, Schambach A, Pfeifer R, Harper C, Mason C, van Til NP. Dogan Y, et al. Among authors: guda s. Mol Ther Methods Clin Dev. 2022 Nov 3;27:464-487. doi: 10.1016/j.omtm.2022.10.017. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36419467 Free PMC article.
Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy.
Brendel C, Negre O, Rothe M, Guda S, Parsons G, Harris C, McGuinness M, Abriss D, Tsytsykova A, Klatt D, Bentler M, Pellin D, Christiansen L, Schambach A, Manis J, Trebeden-Negre H, Bonner M, Esrick E, Veres G, Armant M, Williams DA. Brendel C, et al. Among authors: guda s. Mol Ther Methods Clin Dev. 2020 Mar 17;17:589-600. doi: 10.1016/j.omtm.2020.03.015. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32300607 Free PMC article.
Improving sensitivity of XANES structural fit to the bridged metal-metal coordination.
Abrosimov SV, Protsenko BO, Mannaa AS, Vlasenko VG, Guda SA, Pankin IA, Burlov AS, Koshchienko YV, Guda AA, Soldatov AV. Abrosimov SV, et al. Among authors: guda sa. J Synchrotron Radiat. 2024 May 1;31(Pt 3):447-455. doi: 10.1107/S1600577524002091. Epub 2024 Mar 26. J Synchrotron Radiat. 2024. PMID: 38530834 Free PMC article.
28 results