Antisense oligonucleotide treatment following viral delivery of artificial SOD1-targeting miRNA shows improved efficacy in SOD1-G93A mice.

Zachary C E Hawley¹, Amanda J Guise¹, Ingrid D Pardo¹, Twinkle Chowdhury¹, Sam Hana¹, Maria I Zavodszky¹, Linda Engle¹, Kelly Wiltberger¹, Dan Bartlett¹, Denitza Raitcheva¹, Santhosh Bommegowda¹, Wan-Hung Lee¹, Shukkwan K Chen¹, Adam Sheehy¹, Rachelle Driscoll¹, Michael Peterson¹, Jennifer Sebalusky¹, Stefan Hamann¹, Galina Marsh¹, Jessica Doherty¹, Kay-Ellen Chicoine¹, Paige Cundiff¹, Sean Dwyer¹, Pete Clarner¹, Weike Zeng¹, Daniel Ferretti¹, Taras Tuczkewycz¹, Isabel Isaza¹, Melissa Patrovanie¹, Collin Mcgladrigan¹, Jenhwa Chu¹, James P Morrison², H Moore Arnold¹, Shen Shen¹, Jessica A Hurt¹, Patrick Trapa¹, Danielle L Graham¹, Edward D Plowey¹, Shih-Ching Lo¹

¹Biogen, Cambridge, MA 02142, USA.

²Charles River Laboratories, Shrewsbury, MA 01545, USA.

PubMed PMC DOI PDF

Abstract

Adeno-associated virus (AAV) artificial microRNAs (amiRNAs) targeting superoxide dismutase 1 (SOD1) have been proposed as a therapeutic strategy for people living with amyotrophic lateral sclerosis (ALS) who harbor toxic gain-of-function variants in the SOD1 gene. Clinical efforts have primarily focused on AAV delivery via the cerebrospinal fluid (CSF), as blood-brain-barrier-crossing capsids are…

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