Mammalian ubiquitous promoter isolated from proximal regulatory region of bovine MSTN gene.

Autor: Eom KH; Department of Theriogenology and Biotechnology, College of Veterinary Medicine, Seoul National University, 1 Gwanak-ro, Gwanak-gu, Seoul, 08826, Republic of Korea.; LARTBio Inc., 60 Haan-ro, Gwangmyeong-si, Gyeonggi-do, 14322, Republic of Korea., Kwon DH; Department of Theriogenology and Biotechnology, College of Veterinary Medicine, Seoul National University, 1 Gwanak-ro, Gwanak-gu, Seoul, 08826, Republic of Korea., Kim YC; Department of Theriogenology and Biotechnology, College of Veterinary Medicine, Seoul National University, 1 Gwanak-ro, Gwanak-gu, Seoul, 08826, Republic of Korea., Gim GM; LARTBio Inc., 60 Haan-ro, Gwangmyeong-si, Gyeonggi-do, 14322, Republic of Korea., Yum SY; LARTBio Inc., 60 Haan-ro, Gwangmyeong-si, Gyeonggi-do, 14322, Republic of Korea., Kim SM; College of Pharmacy, Seoul National University, 1 Gwanak-ro, Gwanak-gu, Seoul, 08826, Republic of Korea., Cha HJ; College of Pharmacy, Seoul National University, 1 Gwanak-ro, Gwanak-gu, Seoul, 08826, Republic of Korea., Jang G; Department of Theriogenology and Biotechnology, College of Veterinary Medicine, Seoul National University, 1 Gwanak-ro, Gwanak-gu, Seoul, 08826, Republic of Korea. snujang@snu.ac.kr.; LARTBio Inc., 60 Haan-ro, Gwangmyeong-si, Gyeonggi-do, 14322, Republic of Korea. snujang@snu.ac.kr.; Comparative Medicine Disease Research Center, Seoul National University, 1 Gwanak-ro, Gwanak-gu, Seoul, 08826, Republic of Korea. snujang@snu.ac.kr.
Jazyk: angličtina
Zdroj: Scientific reports [Sci Rep] 2024 Nov 12; Vol. 14 (1), pp. 27750. Date of Electronic Publication: 2024 Nov 12.
DOI: 10.1038/s41598-024-76937-2
Abstrakt: Gene therapy is a promising method for treating inherited diseases by directly delivering the correct genetic material into patient cells. However, the limited packaging capacity of vectors poses a challenge. Minimizing promoter size is a viable strategy among various approaches to address this issue. This study aims to optimize the bovine myostatin (MSTN) promoter, enhancing its utility in gene therapy applications. We identified the primary driver of activity as the proximal regulatory region. Isolated from the native promoter and termed M243, this 243-bp sequence was assessed for its potential as a ubiquitous promoter. In a variety of cell types, including bovine embryos and embryonic stem cells, the M243 promoter showed consistent expression, highlighting its suitability for applications requiring compact promoters. The isolation of a highly conserved, compact 243-bp sequence serving as a promoter suggests a solution for overcoming the size constraints of AAV vectors, suggesting potential contributions to the field of gene therapy.
Competing Interests: Declarations Competing interests The authors declare no competing interests.
(© 2024. The Author(s).)
Databáze: MEDLINE
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