Live-imaging of revertant and therapeutically restored dystrophin in the Dmd

Autor:	M V, Petkova, A, Stantzou, A, Morin, O, Petrova, S, Morales-Gonzalez, F, Seifert, J, Bellec-Dyevre, T, Manoliu, A, Goyenvalle, L, Garcia, I, Richard, C, Laplace-Builhé, M, Schuelke, H, Amthor
Rok vydání:	2019
Předmět:	Gene Editing Mice Transgenic Exons Genetic Therapy Retina Dystrophin Muscular Dystrophy Duchenne Mice Sarcolemma Mice Inbred mdx Animals Humans CRISPR-Cas Systems Alleles
Zdroj:	Neuropathology and applied neurobiologyReferences. 46(6)
ISSN:	1365-2990
Popis:	DmdWe generated a DmdIntravital microscopy in anaesthetized mice allowed live-imaging of sarcolemmal dystrophin-EGFP fusion protein of revertant fibres as well as following therapeutic restoration. Dystrophin-EGFP-fluorescence persisted ex vivo, allowing live-imaging of revertant and therapeutically restored dystrophin in isolated fibres ex vivo. Expression of the shorter dystrophin-EGFP isoforms Dp71 in the brain, Dp260 in the retina, and Dp116 in the peripheral nerve remained unabated by the mdx-23 mutation.Intravital imaging of Dmd
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=pmid________::d6927f25939529ad14f8d845e92a60b5 https://pubmed.ncbi.nlm.nih.gov/32573804 Zobrazit plný text záznamu Plný text