Anti-gene oligonucleotides targeting Friedreich's ataxia expanded GAAâ TTC repeats increase Frataxin expression.
针对弗里德赖希共济失调的 GAAâ‹ TTC 重复序列的抗基因寡核苷酸可增加 Frataxin 表达
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2025.102541
Mozafari Negin, Milagres Salomé, Umek Tea, Rocha Cristina S J, Vargiu Claudia M, Freyberger Fiona, Saher Osama, Napierala Marek, Napierala Jill S, Blomberg Pontus, Jørgensen Per T, Punga Tanel, Smith C I Edvard, Wengel Jesper, Zain Rula
