Safety through design: Expanding options for spinal muscular atrophy gene therapy.
作者信息
Groen Ewout J N, Wadman Renske I
机构信息
Department of Neurology and Neurosurgery, University Medical Center Utrecht, Utrecht, the Netherlands.
出版信息
Mol Ther Methods Clin Dev. 2025 Aug 19;33(3):101550. doi: 10.1016/j.omtm.2025.101550. eCollection 2025 Sep 11.
Abstract
摘要
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3
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4
Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor.
EMBO Mol Med. 2024 Apr;16(4):945-965. doi: 10.1038/s44321-024-00037-x. Epub 2024 Feb 27.
5
Innovating spinal muscular atrophy models in the therapeutic era.
Dis Model Mech. 2023 Sep 1;16(9). doi: 10.1242/dmm.050352. Epub 2023 Oct 3.
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