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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: mason c. 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: mason c. 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: mason c. 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.
Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector.
Dahl M, Smith EMK, Warsi S, Rothe M, Ferraz MJ, Aerts JMFG, Golipour A, Harper C, Pfeifer R, Pizzurro D, Schambach A, Mason C, Karlsson S. Dahl M, et al. Among authors: mason c. Mol Ther Methods Clin Dev. 2020 Dec 3;20:312-323. doi: 10.1016/j.omtm.2020.11.018. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33511245 Free PMC article.
An autonucleolytic suspension HEK293F host cell line for high-titer serum-free AAV5 and AAV9 production with reduced levels of DNA impurity.
Howe G, Bal M, Wasmuth M, Massaro G, Rahim AA, Ali S, Rivera M, Schofield DM, Omotosho A, Ward J, Keshavarz-Moore E, Mason C, Nesbeth DN. Howe G, et al. Among authors: mason c. Mol Ther Methods Clin Dev. 2024 Aug 12;32(3):101317. doi: 10.1016/j.omtm.2024.101317. eCollection 2024 Sep 12. Mol Ther Methods Clin Dev. 2024. PMID: 39257529 Free PMC article.
Cells: from Robert Hooke to cell therapy--a 350 year journey.
Hyllner J, Mason C, Wilmut I. Hyllner J, et al. Among authors: mason c. Philos Trans R Soc Lond B Biol Sci. 2015 Oct 19;370(1680):20150320. doi: 10.1098/rstb.2015.0320. Philos Trans R Soc Lond B Biol Sci. 2015. PMID: 26416687 Free PMC article. No abstract available.
Host metabolism balances microbial regulation of bile acid signalling.
Won TH, Arifuzzaman M, Parkhurst CN, Miranda IC, Zhang B, Hu E, Kashyap S, Letourneau J, Jin WB, Fu Y, Guzior DV; JRI Live Cell Bank; Quinn RA, Guo CJ, David LA, Artis D, Schroeder FC. Won TH, et al. Nature. 2025 Jan 8. doi: 10.1038/s41586-024-08379-9. Online ahead of print. Nature. 2025. PMID: 39779854
2,027 results