Efficacy and safety of a SOD1-targeting artificial miRNA delivered by AAV9 in mice are impacted by miRNA scaffold selection
Titel:
Efficacy and safety of a SOD1-targeting artificial miRNA delivered by AAV9 in mice are impacted by miRNA scaffold selection
Auteur:
Chen, Shukkwan K. Hawley, Zachary C.E. Zavodszky, Maria I. Hana, Sam Ferretti, Daniel Grubor, Branka Hawes, Michael Xu, Shanqin Hamann, Stefan Marsh, Galina Cullen, Patrick Challa, Ravi Carlile, Thomas M. Zhang, Hang Lee, Wan-Hung Peralta, Andrea Clarner, Pete Wei, Cong Koszka, Kathryn Gao, Feng Lo, Shih-Ching