Preclinical assessment of splicing modulation therapy for ABCA4 variant c.768G>T in Stargardt disease
针对 Stargardt 病中 ABCA4 变异 c.768G>T 的剪接调节疗法的临床前评估
期刊:Commun Med (Lond)
影响因子:
doi:10.1038/s43856-024-00712-7
Dyah W Karjosukarso, Femke Bukkems, Lonneke Duijkers, Tomasz Z Tomkiewicz, Julia Kiefmann, Andrei Sarlea, Sander Bervoets, Irene Vázquez-Domínguez, Laurie L Molday, Robert S Molday, Mihai G Netea, Carel B Hoyng, Alejandro Garanto, Rob W J Collin