Amphiphilic lipopeptide significantly enhances uptake of charge-neutral splice switching morpholino oligonucleotide in spinal muscular atrophy patient-derived fibroblasts

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Funding Acknowledgements

B.T.-A. acknowledges award of a University of Melbourne-CSIRO PhD scholarship. The authors wish to thank the Biological Electron Microscopy Facility at CSIRO, particularly Dr Jacinta White for her assistance with Cryo-TEM imaging. We also acknowledge Dr Thomas Meikle (RMIT University) for his assistance with the SAXS experiments. This work was performed in part at the Materials Characterisation and Fabrication Platform (MCFP) and the Victorian Node of the Australian National Fabrication Facility (ANFF) at the University of Melbourne. Work in the laboratory of F.S was supported by the Bethlehem Griffith Research Foundation grant (BGRF1501).