Reactivating the phototransduction cascade with a mutation agnostic gene therapy preserves vision in rod-cone dystrophies
利用不依赖特定基因突变的基因疗法重新激活光转导级联反应,可以保护视杆细胞-视锥细胞营养不良患者的视力。
期刊:iScience
影响因子:4.1
doi:10.1016/j.isci.2025.112106
Simon, Cardillia-Joe; Khabou, Hanen; Chaffiol, Antoine; Rucli, Marco; Finzi, Marion; Norberg, Nat; Grimaud, Anaïs; Mücher, Brix; Desrosiers, Mélissa; Sancho, Serge; Bonilha, Vera Lucia; Grieve, Kate; Duebel, Jens; Paques, Michel; Picaud, Serge; Sahel, José Alain; Audo, Isabelle; Herlitze, Stefan; Dalkara, Deniz