|
|||||
|
|
Proof-of-Concept: Antisense Oligonucleotide Mediated Skipping of Fibrillin-1 Exon 52 |
|
| Authors: | Jessica M. Cale Kane Greer Sue Fletcher Steve D. Wilton |
| Affiliation: | 1.Centre for Molecular Medicine and Innovative Therapeutics, Health Futures Institute, Murdoch University, Murdoch, WA 6150, Australia; (J.M.C.); (K.G.); (S.F.);2.Centre for Neuromuscular and Neurological Disorders, Perron Institute for Neurological and Translational Science, The University of Western Australia, Nedlands, WA 6009, Australia;3.PYC Therapeutics, Nedlands, WA 6009, Australia |
| Abstract: | |
| Keywords: | Marfan syndrome fibrillin-1 antisense oligonucleotides exon skipping splice-switching |