Combination of valproic acid and morpholino splice-switching oligonucleotide produces improved outcomes in spinal muscular atrophy patient-derived fibroblasts

Anna Farrelly-Rosch; Chew Ling Lau; Nitin Patil; Bradley J Turner; Fazel Shabanpoor

Journal article

Combination of valproic acid and morpholino splice-switching oligonucleotide produces improved outcomes in spinal muscular atrophy patient-derived fibroblasts

Anna Farrelly-Rosch, Chew Ling Lau, Nitin Patil, Bradley J Turner, Fazel Shabanpoor

Neurochemistry International | PERGAMON-ELSEVIER SCIENCE LTD | Published : 2017

DOI: 10.1016/j.neuint.2017.02.016

University of Melbourne Researchers

Linda Lau Author Florey Department of Neuroscience and Mental Health

Fazel Shabanpoor Author Florey Department of Neuroscience and Mental Health

Bradley Turner Author Florey Department of Neuroscience and Mental Health

Related Projects (2)

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Currently, there is no known cure for certain neuromuscular genetic disorders. However, recently identified synthetic DNA-type biomolecules ..

SMN IMMUNOGENE THERAPY FOR MOTOR NEURON DISORDERS

We have designed novel gene therapy agents to treat MND. This therapeutic approach uniquely combines gene therapy agents with antibodies whi..

Grants

Awarded by CJ Martin Fellowship from the Australian National Health and Medical Research Council

Awarded by Bethlehem Griffith Research Foundation

Awarded by University of Melbourne

Awarded by Australian National Health and Medical Research Council

Awarded by MND Research Institute of Australia

Funding Acknowledgements

We would like to thank Prof. Philip M Beart for his help with revising the manuscript. FS is a recipient of a CJ Martin Fellowship from the Australian National Health and Medical Research Council (APP1034300) and also acknowledges support by the Bethlehem Griffith Research Foundation (BGRF1501) and support by Early Career Research grant (704-6496, University of Melbourne). Work in the laboratory of BJT was funded by the Australian National Health and Medical Research Council (1104299), Stafford Fox Medical Research Foundation, MND Research Institute of Australia (1609) and Cure for MND Foundation. The Florey Institute of Neuroscience and Mental Health acknowledges the strong support from the Victorian Government and in particular the funding from the Operational Infrastructure Support Grant.