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

Autor: Dunhui Li, Abbie M. Adams, Russell D. Johnsen, Susan Fletcher, Steve D. Wilton

Publikováno v: Molecular Therapy: Nucleic Acids, Vol 22, Iss , Pp 263-272 (2020)

Dystrophin plays a crucial role in maintaining sarcolemma stability during muscle contractions, and mutations that prevent the expression of a functional protein cause Duchenne muscular dystrophy (DMD). Antisense oligonucleotide-mediated manipulation

Externí odkaz: https://doaj.org/article/5194b4eb8cc142648cdf40c35c8296d1

Antisense oligonucleotide induction of progerin in human myogenic cells.

Autor: Yue-Bei Luo, Chalermchai Mitrpant, Abbie M Adams, Russell D Johnsen, Sue Fletcher, Frank L Mastaglia, Steve D Wilton

Publikováno v: PLoS ONE, Vol 9, Iss 6, p e98306 (2014)

We sought to use splice-switching antisense oligonucleotides to produce a model of accelerated ageing by enhancing expression of progerin, translated from a mis-spliced lamin A gene (LMNA) transcript in human myogenic cells. The progerin transcript (

Externí odkaz: https://doaj.org/article/9add1f9fedcb42bf851408035e7bef7b

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

Autor: Helena M. Viola, Sue Fletcher, Abbie M. Adams, Victoria P.A. Johnstone, Livia C. Hool

Publikováno v: JACC: Basic to Translational Science. 3:391-402

Current clinical trials demonstrate Duchenne muscular dystrophy (DMD) patients receiving phosphorodiamidate morpholino oligomer (PMO) therapy exhibit improved ambulation and stable pulmonary function; however, cardiac abnormalities remain. Utilizing

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::ca4bb12046278da513921b16a6e8cde3
https://doi.org/10.1016/j.jacbts.2018.03.007

Altered myogenesis and premature senescence underlie human TRIM32-related myopathy

Autor: Emilia Servián-Morilla, A. L. Pelayo-Negro, Macarena Cabrera-Serrano, Fabiola Mavillard, Nigel G. Laing, Abbie M. Adams, Gianina Ravenscroft, Alejandra Carvajal, M. A. Fernández-García, Sue Fletcher, Jose Luis Nieto-Gonzalez, Carmen Paradas, Reimar Junckerstorff, E. Rivas-Infante, J. M. Dyke, Phillipa J. Lamont

Publikováno v: Acta Neuropathologica Communications
Acta Neuropathologica Communications, Vol 7, Iss 1, Pp 1-16 (2019)
idUS. Depósito de Investigación de la Universidad de Sevilla
instname
Digital.CSIC. Repositorio Institucional del CSIC

TRIM32 is a E3 ubiquitin -ligase containing RING, B-box, coiled-coil and six C-terminal NHL domains. Mutations involving NHL and coiled-coil domains result in a pure myopathy (LGMD2H/STM) while the only described mutation in the B-box domain is assoc

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0aafbf2991c3b2e4dbcb2c6496b47371
http://europepmc.org/articles/PMC6396567

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

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

Publikováno v: Scientific reports, vol 8, iss 1
Scientific Reports
Scientific Reports, Vol 8, Iss 1, Pp 1-16 (2018)

Cell penetrating peptides (CPPs) offer great potential to deliver therapeutic molecules to previously inaccessible intracellular targets. However, many CPPs are inefficient and often leave their attached cargo stranded in the cell’s endosome. We re

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2982281cc58305b13bc410a28e61452e
https://escholarship.org/uc/item/9539k69b