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Gene and Cellular Therapies for Leukodystrophies.
Aerts-Kaya F, van Til NP. Aerts-Kaya F, et al. Among authors: van til np. Pharmaceutics. 2023 Oct 24;15(11):2522. doi: 10.3390/pharmaceutics15112522. Pharmaceutics. 2023. PMID: 38004502 Free PMC article. Review.
Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning.
Huston MW, van Til NP, Visser TP, Arshad S, Brugman MH, Cattoglio C, Nowrouzi A, Li Y, Schambach A, Schmidt M, Baum C, von Kalle C, Mavilio F, Zhang F, Blundell MP, Thrasher AJ, Verstegen MM, Wagemaker G. Huston MW, et al. Among authors: van til np. Mol Ther. 2011 Oct;19(10):1867-77. doi: 10.1038/mt.2011.127. Epub 2011 Jul 12. Mol Ther. 2011. PMID: 21750532 Free PMC article.
45 results