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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. Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor. EMBO Mol Med. 2024 Feb 27.

View in: PubMed