PPMO-based exon skipping therapy improves respiratory function in the mdx mouse model of Duchenne muscular dystrophy.
基于 PPMO 的外显子跳跃疗法可改善杜氏肌营养不良症 mdx 小鼠模型的呼吸功能。
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2025.102810
Biswas Debolina D, Rodriguez Maran Y Hernandez, El Haddad Lea, Hoffmann Gwendolyn, Copson Elena, Mayman Sloane, Jose Julia, Lang Sydney, Chen Olivia, Dela Pena Andrea, Levin Emerson, Abdelbarr Mariam, Slyne Aoife, ElMallah Mai K
