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 |