Systemic antibody-oligonucleotide delivery to the central nervous system ameliorates mouse models of spinal muscular atrophy
Suzan M. Hammond, Frank Abendroth, Larissa Goli, Matthew R. Burrell, George Thom, Ian K. Gurrell, Jessica Stoodley, Nina Ahlskog, Michael John Gait, Matthew J. A. Wood, Carl I. Webster
This study demonstrates that systemic delivery of ASO conjugated to a brain-targeting antibody improves SMN2 splicing and survival in SMA mouse models.
bioRxiv · Jul 29, 2021 · PreprintMolecular Biology