Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agents

por Wilton, Steve D, Fletcher, Sue
Publicado 2011

Regulation of eukaryotic gene expression by the untranslated gene regions and other non-coding elements

por Barrett, Lucy W., Fletcher, Sue, Wilton, Steve D.
Publicado 2012

Breakpoint junction features of seven DMD deletion mutations

por Keegan, Niall P., Wilton, Steve D., Fletcher, Sue
Publicado 2019

Analysis of Pathogenic Pseudoexons Reveals Novel Mechanisms Driving Cryptic Splicing

por Keegan, Niall P., Wilton, Steve D., Fletcher, Sue
Publicado 2022

Corrigendum: Analysis of Pathogenic Pseudoexons Reveals Novel Mechanisms Driving Cryptic Splicing

por Keegan, Niall P., Wilton, Steve D., Fletcher, Sue
Publicado 2022

Antisense-mediated splice intervention to treat human disease: the odyssey continues

por Pitout, Ianthe, Flynn, Loren L., Wilton, Steve D., Fletcher, Sue
Publicado 2019

Progress in the molecular pathogenesis and nucleic acid therapeutics for Parkinson's disease in the precision medicine era

por Li, Dunhui, Mastaglia, Frank L., Fletcher, Sue, Wilton, Steve D.
Publicado 2020

Proof-of-Concept: Antisense Oligonucleotide Mediated Skipping of Fibrillin-1 Exon 52

por Cale, Jessica M., Greer, Kane, Fletcher, Sue, Wilton, Steve D.
Publicado 2021

Polyglutamine Ataxias: Our Current Molecular Understanding and What the Future Holds for Antisense Therapies

por McIntosh, Craig S., Li, Dunhui, Wilton, Steve D., Aung-Htut, May T.
Publicado 2021

Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene

por Greer, Kane L, Lochmüller, Hanns, Flanigan, Kevin, Fletcher, Susan, Wilton, Steve D
Publicado 2014

Evaluation of exon-skipping strategies for Duchenne muscular dystrophy utilizing dystrophin-deficient zebrafish

por Berger, Joachim, Berger, Silke, Jacoby, Arie S, Wilton, Steve D, Currie, Peter D
Publicado 2011

Functional improvement of dystrophic muscle by repression of utrophin: let-7c interaction

por Mishra, Manoj K., Loro, Emanuele, Sengupta, Kasturi, Wilton, Steve D., Khurana, Tejvir S.
Publicado 2017

Removal of the Polyglutamine Repeat of Ataxin-3 by Redirecting pre-mRNA Processing

por McIntosh, Craig S., Aung-Htut, May Thandar, Fletcher, Sue, Wilton, Steve D.
Publicado 2019

Nonsequential Splicing Events Alter Antisense-Mediated Exon Skipping Outcome in COL7A1

por Ham, Kristin A., Aung-Htut, May Thandar, Fletcher, Sue, Wilton, Steve D.
Publicado 2020

Splice correction therapies for familial hypercholesterolemic patients with low-density lipoprotein receptor mutations

por McIntosh, Craig S., Watts, Gerald F., Wilton, Steve D., Aung-Htut, May T.
Publicado 2021

Translational Regulation of Utrophin by miRNAs

por Basu, Utpal, Lozynska, Olga, Moorwood, Catherine, Patel, Gopal, Wilton, Steve D., Khurana, Tejvir S.
Publicado 2011

Rational Design of Short Locked Nucleic Acid-Modified 2′-O-Methyl Antisense Oligonucleotides for Efficient Exon-Skipping In Vitro

por Le, Bao T., Adams, Abbie M., Fletcher, Susan, Wilton, Stephen D., Veedu, Rakesh N.
Publicado 2017

Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN2 Transcript

por Flynn, Loren L., Mitrpant, Chalermchai, Pitout, Ianthe L., Fletcher, Sue, Wilton, Steve D.
Publicado 2018

Single Exon Skipping Can Address a Multi-Exon Duplication in the Dystrophin Gene

por Greer, Kane, Johnsen, Russell, Nevo, Yoram, Fellig, Yakov, Fletcher, Susan, Wilton, Steve D.
Publicado 2020

Morpholino Oligomer-Induced Dystrophin Isoforms to Map the Functional Domains in the Dystrophin Protein

por Li, Dunhui, Adams, Abbie M., Johnsen, Russell D., Fletcher, Susan, Wilton, Steve D.
Publicado 2020