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Page 1
Gene Therapy Developments for Pompe Disease.
Unnisa Z, Yoon JK, Schindler JW, Mason C, van Til NP. Unnisa Z, et al. Among authors: yoon jk. Biomedicines. 2022 Jan 28;10(2):302. doi: 10.3390/biomedicines10020302. Biomedicines. 2022. PMID: 35203513 Free PMC article. Review.
High-throughput analysis of hematopoietic stem cell engraftment after intravenous and intracerebroventricular dosing.
Plasschaert RN, DeAndrade MP, Hull F, Nguyen Q, Peterson T, Yan A, Loperfido M, Baricordi C, Barbarossa L, Yoon JK, Dogan Y, Unnisa Z, Schindler JW, van Til NP, Biasco L, Mason C. Plasschaert RN, et al. Among authors: yoon jk. Mol Ther. 2022 Oct 5;30(10):3209-3225. doi: 10.1016/j.ymthe.2022.05.022. Epub 2022 May 25. Mol Ther. 2022. PMID: 35614857 Free PMC article.
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: yoon jk. 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 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. 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
326 results