Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models
剪接调节寡核苷酸 QR-110 可恢复人类 c.2991+1655A>G LCA10 模型中的 CEP290 mRNA 和功能
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.5
doi:10.1016/j.omtn.2018.07.010
Kalyan Dulla, Monica Aguila, Amelia Lane, Katarina Jovanovic, David A Parfitt, Iris Schulkens, Hee Lam Chan, Iris Schmidt, Wouter Beumer, Lars Vorthoren, Rob W J Collin, Alejandro Garanto, Lonneke Duijkers, Anna Brugulat-Panes, Ma'ayan Semo, Anthony A Vugler, Patricia Biasutto, Peter Adamson, Michae