http://dspace.bsu.edu.ru/handle/123456789/47937
Название: | Therapeutic potential of highly functional codon‑optimized microutrophin for muscle‑specific expression |
Авторы: | Starikova, A. V. Skopenkova, V. V. Polikarpova, A. V. Reshetov, D. A. Soldatov, V. O. |
Ключевые слова: | medicine medical genetics gene therapy muscle‑specific expression rAAV9-μUtrn expression microutrophin muscular dystrophy |
Дата публикации: | 2022 |
Библиографическое описание: | Therapeutic potential of highly functional codon‑optimized microutrophin for muscle‑specific expression / A.V. Starikova [et al.] // Scientific Reports. - 2022. - Vol.12-Art. 848. - URL: https://www.nature.com/articles/s41598-022-04892-x.pdf |
Краткий осмотр (реферат): | High expectations have been set on gene therapy with an AAV-delivered shortened version of dystrophin (μDys) for Duchenne muscular dystrophy (DMD), with several drug candidates currently undergoing clinical trials. Safety concerns with this therapeutic approach include the immune response to introduced dystrophin antigens observed in some DMD patients. Recent reports highlighted microutrophin (μUtrn) as a less immunogenic functional dystrophin substitute for gene therapy. In the current study, we created a human codon-optimized μUtrn which was subjected to side-by-side characterization with previously reported mouse and human μUtrn sequences after rAAV9 intramuscular injections in mdx mice |
URI (Унифицированный идентификатор ресурса): | http://dspace.bsu.edu.ru/handle/123456789/47937 |
Располагается в коллекциях: | Статьи из периодических изданий и сборников (на иностранных языках) = Articles from periodicals and collections (in foreign languages) |
Файл | Описание | Размер | Формат | |
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Soldatov_Therapeutic.pdf | 2.62 MB | Adobe PDF | Просмотреть/Открыть |
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