People

Prof Steve Wilton, C/Prof Allan Kermode, Dr Rakesh Veedu, Prof Sue Fletcher and Dr May Thandar Aung-Htut received $50,000 from MS Research Australia for “Blocking genes to treat MS”.

Flynn LL, Mitrpant C, Pitout IL, Fletcher S, and Wilton SD, Antisense oligonucleotide mediated terminal intron retention of the SMN2 transcript. Mol Ther Nucleic Acids, 2018. (in press).

Martinovich KM, Shaw NC, Kicic A, Schultz A, Fletcher S, Wilton SD, and Stick SM, The potential of antisense oligonucleotide therapies for inherited childhood lung diseases. Mol Cell Pediatr, 2018. 5(1): p. 3.

Viola HM, Johnstone VPA, Adams AM, Fletcher S, and Hool LC, A Morpholino Oligomer Therapy Regime That Restores Mitochondrial Function and Prevents mdx Cardiomyopathy. JACC Basic Transl Sci, 2018. 3(3): p. 391-402.

Bellgard MI, Napier KR, Bittles AH, Szer J, Fletcher S, Zeps N, Hunter AA, and Goldblatt J, Design of a framework for the deployment of collaborative independent rare disease-centric registries: Gaucher disease registry model. Blood Cells Mol Dis, 2018. 68: p. 232-238.

Flynn LL, Mitrpant C, Pitout IL, Fletcher S, and Wilton SD, Antisense oligonucleotide mediated terminal intron retention of the SMN2 transcript. Mol Ther Nucleic Acids, 2018. (in press).

Martinovich KM, Shaw NC, Kicic A, Schultz A, Fletcher S, Wilton SD, and Stick SM, The potential of antisense oligonucleotide therapies for inherited childhood lung diseases. Mol Cell Pediatr, 2018. 5(1): p. 3.

Sarepta Therapeutics Contract Research Correlation study: PMO relative activity ranking in DMD patient myoblasts and normal myoblasts US$1,405,350

NHMRC European Union RD-CONNECT: An integrated platform connecting registries, biobanks and clinical bioinformatics for rare disease research $989,884

MDA USA Oligomer design & validation for DMD: quantum improvements in exon skipping US$300,000