RNA-Based Therapy Utilizing Oculopharyngeal Muscular Dystrophy Transcript Knockdown and Replacement
利用眼咽肌营养不良症转录本敲低和替换的RNA疗法
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2019.02.003
Abu-Baker, Aida; Kharma, Nawwaf; Perreault, Jonathan; Grant, Alanna; Shekarabi, Masoud; Maios, Claudia; Dona, Michele; Neri, Christian; Dion, Patrick A; Parker, Alex; Varin, Luc; Rouleau, Guy A