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Table 1 Silencing mechanism, nucleic acid species, and muscle delivery systems described in session 6 of the FSHD IRC meeting. aRef 19. bRef 20. cRef 18. dUsed FM10, a previously published sequence targeting the DUX4 polyA signal. Although the PMO operates via steric hindrance, blocking polyadenylation could lead to DUX4 mRNA instability and degradation (Ref 18)

From: Meeting report: the 2021 FSHD International Research Congress

Speaker, affiliation DUX4 silencing mechanism Antisense species Conjugate/muscle delivery system
Katelyn Daman, University of Massachusetts RNAi siRNA Docosanoic acid
Barbora Malecova, Avidity Biosciences RNAi siRNA Murine TfR mAb
Jonathan Van Dyke, Arrowhead RNAi siRNA Not specified
Lindsay Wallace, Nationwide Children’s Hospitala,b RNAi In vivo expressed microRNA AAV6 and AAV9 gene therapy vectors
Linde Bouwman, Leiden University Medical Center RNAse H Gapmer ASO Palmitate (C16)
Ngoc Lu-Nguyen, Royal Holloway Universityc Steric hindrance PMO Vivo-PMO, cell penetrating guanidinium dendrimer
Nelson Hsia, Dyne Steric hindranced PMO TfR antibody Fab fragment