Open Access

Application of urine cells in drug intervention for spinal muscular atrophy

  • Authors:
    • Qi‑Jie Zhang
    • Xiang Lin
    • Jin‑Jing Li
    • Ying‑Qian Lu
    • Xin‑Xin Guo
    • En‑Lin Dong
    • Miao Zhao
    • Jin He
    • Ning Wang
    • Wan‑Jin Chen
  • View Affiliations

  • Published online on: July 12, 2017     https://doi.org/10.3892/etm.2017.4791
0

Abstract

Spinal muscular atrophy (SMA) is a lethal childhood neurodegenerative disorder that is caused by the homozygous deletion of survival motor neuron 1 (SMN1). To date, no effective treatments are available. In the current study, urine cells taken from SMA patients were cultured and the application of patient‑derived urine cells was determined in drug intervention. A total of 13 SMA patient‑derived urine cell lines and 40 control cell lines were established. SMN was highly expressed in the nucleus and cytoplasm. Patient‑derived urine cells expressed low levels of SMN protein compared with controls, they exhibited good tolerance to chemical and electrical damage. SMN expression was upregulated following treatment with histone deacetylase inhibitors and the effect was greater in groups treated with morpholino modified antisense oligo, which targets ISS‑N1 in SMN2 intron 7. The results of the current study indicated that SMA patient‑derived urine cells may be useful in the initial screening of potential compounds and drugs to treat SMA.

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Print ISSN: 1792-0981
Online ISSN:1792-1015

2016 Impact Factor: 1.261
Ranked #50/128 Medicine Research and Experimental
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APA
Zhang, Q., Lin, X., Li, J., Lu, Y., Guo, X., Dong, E. ... Chen, W. (1899). Application of urine cells in drug intervention for spinal muscular atrophy. Experimental and Therapeutic Medicine, 0, 0-0. https://doi.org/10.3892/etm.2017.4791
MLA
Zhang, Q., Lin, X., Li, J., Lu, Y., Guo, X., Dong, E., Zhao, M., He, J., Wang, N., Chen, W."Application of urine cells in drug intervention for spinal muscular atrophy". Experimental and Therapeutic Medicine 0.0 (1899): 0-0.
Chicago
Zhang, Q., Lin, X., Li, J., Lu, Y., Guo, X., Dong, E., Zhao, M., He, J., Wang, N., Chen, W."Application of urine cells in drug intervention for spinal muscular atrophy". Experimental and Therapeutic Medicine 0, no. 0 (1899): 0-0. https://doi.org/10.3892/etm.2017.4791