{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T08:06:17Z","timestamp":1790841977794,"version":"4.1.0"},"reference-count":0,"publisher":"Acta Naturae Ltd","issue":"1","license":[{"start":{"date-parts":[[2021,3,15]],"date-time":"2021-03-15T00:00:00Z","timestamp":1615766400000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Acta Naturae"],"abstract":"<jats:p>Many genetic diseases that are responsible for muscular disorders have been described to date. Gene replacement therapy is a state-of-the-art strategy used to treat such diseases. In this approach, the functional copy of a gene is delivered to the affected tissues using viral vectors. There is an urgent need for the design of short, regulatory sequences that would drive a high and robust expression of a therapeutic transgene in skeletal muscles, the diaphragm, and the heart, while exhibiting limited activity in non-target tissues. This review focuses on the development and improvement of muscle-specific promoters based on skeletal muscle -actin, muscle creatine kinase, and desmin genes, as well as other genes expressed in muscles. The current approaches used to engineer synthetic muscle-specific promoters are described. Other elements of the viral vectors that contribute to tissue-specific expression are also discussed. A special feature of this review is the presence of up-to-date information on the clinical and preclinical trials of gene therapy drug candidates that utilize muscle-specific promoters.<\/jats:p>","DOI":"10.32607\/actanaturae.11063","type":"journal-article","created":{"date-parts":[[2021,4,21]],"date-time":"2021-04-21T09:24:38Z","timestamp":1618997078000},"page":"47-58","source":"Crossref","is-referenced-by-count":58,"title":["Muscle-Specific Promoters for Gene Therapy"],"prefix":"10.32607","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7267-7967","authenticated-orcid":false,"given":"Victoria V.","family":"Skopenkova","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tatiana V.","family":"Egorova","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maryana V.","family":"Bardina","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"17808","published-online":{"date-parts":[[2021,3,15]]},"container-title":["Acta Naturae"],"original-title":[],"link":[{"URL":"https:\/\/actanaturae.ru\/2075-8251\/article\/viewFile\/11063\/pdf_1","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/actanaturae.ru\/2075-8251\/article\/viewFile\/11063\/pdf_1","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,11,15]],"date-time":"2021-11-15T13:47:55Z","timestamp":1636984075000},"score":1,"resource":{"primary":{"URL":"https:\/\/actanaturae.ru\/2075-8251\/article\/view\/11063"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,3,15]]},"references-count":0,"journal-issue":{"issue":"1","published-online":{"date-parts":[[2021,3,15]]}},"URL":"https:\/\/doi.org\/10.32607\/actanaturae.11063","relation":{},"ISSN":["2075-8251"],"issn-type":[{"value":"2075-8251","type":"print"}],"subject":[],"published":{"date-parts":[[2021,3,15]]}}}