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Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease.
Jeyakumar JM, Kia A, Tam LCS, McIntosh J, Spiewak J, Mills K, Heywood W, Chisari E, Castaldo N, Verhoef D, Hosseini P, Kalcheva P, Cocita C, Miranda CJ, Canavese M, Khinder J, Rosales C, Hughes D, Sheridan R, Corbau R, Nathwani A. Jeyakumar JM, et al. Among authors: kia a. Gene Ther. 2023 Jun;30(6):487-502. doi: 10.1038/s41434-022-00381-y. Epub 2023 Jan 11. Gene Ther. 2023. PMID: 36631545 Free PMC article.