The power to target muscle to
stop or reverse disease progression
On World FSHD Day and every day, Dyne is dedicated to making a difference by advancing potentially life-transforming therapies for individuals and families living with FSHD.
Scientific Publications & Presentations
May 14, 2021
Splice Correction and Reduction of Toxic DMPK RNA In Vitro and In Vivo Utilizing Novel Antibody Targeted Antisense Oligonucleotides
Presented at American Society of Gene & Cell Therapy Annual Meeting (ASGCT), May 14, 2021
May 14, 2021
The FORCE™ Platform Achieves Robust Knock Down of Toxic Human Nuclear DMPK RNA and Foci Reduction in DM1 Cells and in Newly Developed hTfR1/DMSXL Mouse Model
Presented at American Society of Gene & Cell Therapy Annual Meeting (ASGCT), May 14, 2021
May 12, 2020
Targeted Delivery of ASOs Demonstrates Potential to Treat Duchenne Muscular Dystrophy
Presented at ASGCT 2020
Supporting Literature
Platform
Development of Antibody-siRNA Conjugate Targeted to Cardiac and Skeletal Muscles
Journal of Controlled Release
DM1
Identification and Characterization of Modified Antisense Oligonucleotides Targeting DMPK in Mice and Nonhuman Primates for the Treatment of Myotonic Dystrophy Type 1
Nucleic Acids Research (NAR)
In Situ Fluorescence Analysis Demonstrates Active siRNA Exclusion From the Nucleus by Exportin 5
Nucleic Acids Research (NAR)
RNase H1-Dependent Antisense Oligonucleotides Are Robustly Active in Directing RNA Cleavage in Both the Cytoplasm and the Nucleus
Molecular Therapy
The Current State and Future Directions of RNAi-based Therapeutics
Nature Reviews Drug Discovery
Quantitative Fluorescence Imaging Determines the Absolute Number of Locked Nucleic Acid Oligonucleotides Needed for Suppression of Target Gene Expression
Nucleic Acids Research (NAR)
Cellular Localization of Long Non-Coding RNAs Affects Silencing by RNAi More Than by Antisense Oligonucleotides
Nucleic Acids Research (NAR)
RNA-mediated Therapies in Myotonic Dystrophy
Drug Discovery Today
