PRMT inhibitor promotes SMN2 exon 7 inclusion and synergizes with nusinersen to rescue SMA mice.
Journal article
Kordala AJ. et al, (2023), EMBO Mol Med, 15
Application of Antisense Conjugates for the Treatment of Myotonic Dystrophy Type 1.
Journal article
Stoodley J. et al, (2023), Int J Mol Sci, 24
Peptide-Conjugated PMOs for the Treatment of Myotonic Dystrophy.
Journal article
Stoodley J. et al, (2023), Methods Mol Biol, 2587, 209 - 237
Antibody-oligonucleotide conjugate achieves CNS delivery in animal models for spinal muscular atrophy.
Journal article
Hammond SM. et al, (2022), JCI Insight, 7
Evaluating Efficacy of Peptide-Delivered Oligonucleotides Using the Severe Taiwanese SMA Mouse Model.
Chapter
Goli L. et al, (2022), 2383, 491 - 513
Mesyl Phosphoramidate Oligonucleotides as Potential Splice-Switching Agents: Impact of Backbone Structure on Activity and Intracellular Localization.
Journal article
Hammond SM. et al, (2021), Nucleic Acid Ther, 31, 190 - 200