The power to target muscle to

stop or reverse disease progression

Scientific Publications & Presentations

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

Metabolic Catastrophe in Mice Lacking Transferrin Receptor in Muscle EBio Medicine

DM1

Targeting Nuclear RNA for In Vivo Correction of Myotonic Dystrophy Nature

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)

RNAi Factors Are Present and Active in Human Cell Nuclei Cell Reports

RNA-mediated Therapies in Myotonic Dystrophy Drug Discovery Today

DMPK Gene Deletion or Antisense Knockdown Does Not Compromise Cardiac or Skeletal Muscle Function in Mice Human Molecular Genetics

FSHD

Morpholino-Mediated Knockdown of DUX4 Toward Facioscapulohumeral Muscular Dystrophy Therapeutics Molecular Therapy

Antisense Oligonucleotides Used to Target the DUX4 mRNA as Therapeutic Approaches in Facioscapulohumeral Muscular Dystrophy (FSHD) Genes