Journal: Molecular Therapy
Article Title: Cyclic Peptides to Improve Delivery and Exon Skipping of Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy
doi: 10.1016/j.ymthe.2017.10.004
Figure Lengend Snippet: Evaluation of CyPep10-AON Conjugate CyPep10 was conjugated to an AON targeting human dystrophin exon 45 (h45AON and CyPep10-h45AON) to evaluate whether the conjugation of a cyclic peptide has any influence on the exon-skipping functionality of the AON. (A) Human control myotubes incubated with 2 or 4 μM AON without any transfection reagent for 96 hr. Results show the average exon-skipping levels of two independent experiments in duplo. (B) IM injection in two hDMD mice (pretreated with cardiotoxin) with 20 μg of h45AON or molar equivalent of CyPep10-h45AON for 2 consecutive days. Results represent an average of two independent experiments in duplo. Bars represent mean ± SD. G, gastrocnemius; Tr, triceps.
Article Snippet: Subsequently, slides were stained overnight at 4°C with primary antibody (Dystrophin antibody, 1:50 [Santa Cruz, Germany]; sc-7461; Laminin antibody, 1:100 [Abcam, UK]; ab11575) in blocking buffer (PBS/0.05% Tween/5% horse serum).
Techniques: Conjugation Assay, Control, Incubation, Transfection, Injection