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An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs.
Vasireddy V, Maguire CA, Anderson DW, Ng C, Gong Y, Eichler F, Fourcade S, Guilera C, Onieva A, Sanchez A, Leal-Julià M, Verdés S, Dijkstra IME, Kemp S, Park H, Lutz T, Clark SW, Bosch A, Pujol A, Kozarsky K. Vasireddy V, et al. Among authors: onieva a. Mol Ther Methods Clin Dev. 2024 Oct 9;32(4):101354. doi: 10.1016/j.omtm.2024.101354. eCollection 2024 Dec 12. Mol Ther Methods Clin Dev. 2024. PMID: 39524975 Free PMC article.