QR-421a, an antisense oligonucleotide, for the treatment of retinitis pigmentosa due to USH2A exon 13 mutations
色素性视网膜炎
外显子
寡核苷酸
遗传学
生物
突变
基因
作者
Hester van Diepen,Kalyan Dulla,Hee Lam Chan,Iris A. Schulkens,Wouter Beumer,Lars Vorthoren,Cathaline den Besten,Levi C.M. Buil,Janne J. Turunen,Jiayi Miao,Sanne Broekman,Erik de Vrieze,Margo Dona,Sílvia Albert,Erwin van Wijk,Peter Adamson