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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: schindler jw. 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: schindler jw. 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: schindler jw. 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.
Inducible histone K-to-M mutations are dynamic tools to probe the physiological role of site-specific histone methylation in vitro and in vivo.
Brumbaugh J, Kim IS, Ji F, Huebner AJ, Di Stefano B, Schwarz BA, Charlton J, Coffey A, Choi J, Walsh RM, Schindler JW, Anselmo A, Meissner A, Sadreyev RI, Bernstein BE, Hock H, Hochedlinger K. Brumbaugh J, et al. Among authors: schindler jw. Nat Cell Biol. 2019 Nov;21(11):1449-1461. doi: 10.1038/s41556-019-0403-5. Epub 2019 Oct 28. Nat Cell Biol. 2019. PMID: 31659274 Free PMC article.