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

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

Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching

por Fragall, Clayton T, Adams, Abbie M, Johnsen, Russell D, Kole, Ryszard, Fletcher, Sue, Wilton, Steve D
Publicado 2011

A Morpholino Oligomer Therapy Regime That Restores Mitochondrial Function and Prevents mdx Cardiomyopathy

por Viola, Helena M., Johnstone, Victoria P.A., Adams, Abbie M., Fletcher, Susan, Hool, Livia C.
Publicado 2018

Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries

por Adams, Abbie M, Harding, Penny L, Iversen, Patrick L, Coleman, Catherine, Fletcher, Sue, Wilton, Steve D
Publicado 2007

Targeted SMN Exon Skipping: A Useful Control to Assess In Vitro and In Vivo Splice-Switching Studies

por Flynn, Loren L., Mitrpant, Chalermchai, Adams, Abbie, Pitout, Ianthe L., Stirnweiss, Anja, Fletcher, Sue, Wilton, Steve D.
Publicado 2021

Multiple exon skipping strategies to by-pass dystrophin mutations

por Adkin, Carl F., Meloni, Penelope L., Fletcher, Susan, Adams, Abbie M., Muntoni, Francesco, Wong, Brenda, Wilton, Steve D.
Publicado 2012

Antisense Oligonucleotide Induction of Progerin in Human Myogenic Cells

por Luo, Yue-Bei, Mitrpant, Chalermchai, Adams, Abbie M., Johnsen, Russell D., Fletcher, Sue, Mastaglia, Frank L., Wilton, Steve D.
Publicado 2014

Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies

por Toh, Zhi Yon Charles, Thandar Aung-Htut, May, Pinniger, Gavin, Adams, Abbie M., Krishnaswarmy, Sudarsan, Wong, Brenda L., Fletcher, Sue, Wilton, Steve D.
Publicado 2016

Phenotype–genotype correlations in a pseudodominant Stargardt disease pedigree due to a novel ABCA4 deletion–insertion variant causing a splicing defect

por Huang, Di, Thompson, Jennifer A., Charng, Jason, Chelva, Enid, McLenachan, Samuel, Chen, Shang‐Chih, Zhang, Dan, McLaren, Terri L., Lamey, Tina M., Constable, Ian J., De Roach, John N., Aung‐Htut, May Thandar, Adams, Abbie, Fletcher, Sue, Wilton, Steve D., Chen, Fred K.
Publicado 2020

A platform for discovery of functional cell-penetrating peptides for efficient multi-cargo intracellular delivery

por Hoffmann, Katrin, Milech, Nadia, Juraja, Suzy M., Cunningham, Paula T., Stone, Shane R., Francis, Richard W., Anastasas, Mark, Hall, Clinton M., Heinrich, Tatjana, Bogdawa, Heique M., Winslow, Scott, Scobie, Marie N., Dewhurst, Robert E., Florez, Laura, Ong, Ferrer, Kerfoot, Maria, Champain, Danie, Adams, Abbie M., Fletcher, Susan, Viola, Helena M., Hool, Livia C., Connor, Theresa, Longville, Brooke A. C., Tan, Yew-Foon, Kroeger, Karen, Morath, Volker, Weiss, Gregory A., Skerra, Arne, Hopkins, Richard M., Watt, Paul M.
Publicado 2018