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Ribozyme-activated mRNA trans-ligation enables large gene delivery to treat muscular dystrophies.
Science. 2024 Nov 15;386(6723):762-767. doi: 10.1126/science.adp8179. Epub 2024 Nov 14.
Science. 2024.
PMID: 39541470
Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor.
Xie Q, Chen X, Ma H, Zhu Y, Ma Y, Jalinous L, Cox GF, Weaver F, Yang J, Kennedy Z, Gruntman A, Du A, Su Q, He R, Tai PW, Gao G, Xie J.
Xie Q, et al. Among authors: jalinous l.
EMBO Mol Med. 2024 Apr;16(4):945-965. doi: 10.1038/s44321-024-00037-x. Epub 2024 Feb 27.
EMBO Mol Med. 2024.
PMID: 38413838
Free PMC article.
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