Selection and identification of a novel ssDNA aptamer targeting human skeletal muscle

Shuming Sun; Han Liu; Yan Hu; Yanpeng Wang; Mingri Zhao; Yijun Yuan; Yafei Han; Yingying Jing; Jin Cui; Xiaoxiang Ren; Xiao Chen; Jiacan Su

doi:10.1016/j.bioactmat.2022.05.016

Selection and identification of a novel ssDNA aptamer targeting human skeletal muscle

Bioact Mater. 2022 May 27:20:166-178. doi: 10.1016/j.bioactmat.2022.05.016. eCollection 2023 Feb.

Authors

Shuming Sun¹, Han Liu², Yan Hu², Yanpeng Wang¹, Mingri Zhao³, Yijun Yuan⁴, Yafei Han², Yingying Jing², Jin Cui⁵, Xiaoxiang Ren², Xiao Chen⁵, Jiacan Su^{2

5}

Affiliations

¹ Molecular Biology Research Center, Center for Medical Genetics, Hunan Province Key Laboratory of Basic and Applied Hematology, School of Life Sciences, Central South University, Changsha, 410078, China.
² Institute of Translational Medicine, Shanghai University, Shanghai, 200444, China.
³ Center for Medical Genetics & Hunan Key Laboratory of Medical Genetics, School of Life Sciences, Central South University, Changsha, Hunan, 410013, China.
⁴ Molecular Science and Biomedicine Laboratory (MBL), State Key Laboratory of Chemo/Biosensing and Chemometrics, College of Biology, College of Chemistry and Chemical Engineering, Aptamer Engineering Center of Hunan Province, Hunan University, Changsha, Hunan, 410082, China.
⁵ Department of Orthopedics Trauma, Shanghai Changhai Hospital, Naval Medical University, Shanghai, 200433, China.

Abstract

Skeletal muscle disorders have posed great threats to health. Selective delivery of drugs and oligonucleotides to skeletal muscle is challenging. Aptamers can improve targeting efficacy. In this study, for the first time, the human skeletal muscle-specific ssDNA aptamers (HSM01, etc.) were selected and identified with Systematic Evolution of Ligands by Exponential Enrichment (SELEX). The HSM01 ssDNA aptamer preferentially interacted with human skeletal muscle cells in vitro. The in vivo study using tree shrews showed that the HSM01 ssDNA aptamer specifically targeted human skeletal muscle cells. Furthermore, the ability of HSM01 ssDNA aptamer to target skeletal muscle cells was not affected by the formation of a disulfide bond with nanoliposomes in vitro or in vivo, suggesting a potential new approach for targeted drug delivery to skeletal muscles via liposomes. Therefore, this newly identified ssDNA aptamer and nanoliposome modification could be used for the treatment of human skeletal muscle diseases.

Keywords: Aptamer; Human skeletal muscle; Nanoliposomes; SELEX; Target delivery.