AAV2-mediated intravitreal delivery of exon-specific U1 snRNA rescues optic neuropathy in familial dysautonomia
AAV2介导的玻璃体内递送外显子特异性U1 snRNA可挽救家族性自主神经功能障碍患者的视神经病变
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2025.102788
Chekuri, Anil; Kondabolu, Krishnakanth; Kirchner, Emily G; Koli, Swanand; Chagnon, Matthew; Krasniqi-Vanmeter, Drenushe; Stern, Max E; Bolduc, Jessica; Romano, Giulia; Pagani, Franco; Vandenberghe, Luk H; Morini, Elisabetta; Slaugenhaupt, Susan A