Molecular Therapies Research

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.

Inherited retinal disease therapy targeting precursor messenger ribonucleic acid Huang D, Fletcher S, Wilton SD, McLenachan S, Mackey DA, Chen FKVision (2017) in press

Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach Wein N, Vulin A, Findlay AR, Gumienny F, Huang N, Wilton SD, Flanigan KM 2017 Journal Neuromuscul Dis 4: 199-207

Restoration of Cftr Function by Antisense Oligonucleotide Splicing Modulation Oren Y, Tur-Sinai MI, Ozeri-Galai E, Avizur O, Mutyam V, Wilton SD, Rowe SM, Kerem B 2017 Pediatric Pulmonology 52: S313-S313

Response to “Railroading at the FDA” Muntoni F, Fletcher S and Wilton SD 2017 Nat Biotechnol 35: 207-209

Functional improvement of dystrophic muscle by repression of utrophin: let-7c interaction Mishra MK, Loro E, Sengupta K, Wilton SD, Khurana TS 2017 PLoS One 12: e0182676

Corrigendum: Response to “Railroading at the FDA”. Letter to the editor. SD Wilton, S Fletcher and F Muntoni. Nature Biotechnology. (2017) DOI: 10.1038/nbt0517-481d

Translational development of splice-modifying antisense oligomers Fletcher S, Bellgard MI, Price L, Akkari AP, Wilton SD 2017 Expert Opin Biol Ther 17: 15-30

A Dominant-Negative COL6A1 Pseudoexon Insertion Is Skippable Using Splice-Modulating Oligonucleotides Bolduc V, Foley AR, Donkervoort S, Hu Y, Cummings BB, Lek M, Sarathy A, Sizov K, Degefa HS, Wagener R, Hennig GW, Hanssen E, Lamande SR, Muntoni F, Wilton SD, Macarthur DG, Bonnemann CG 2017 Molecular Therapy 25: 119-120