Reactivation of FMR1 gene expression is a promising strategy for fragile X syndrome therapy
Author:
Funder
Russian Foundation for Basic Research
Publisher
Springer Science and Business Media LLC
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
http://www.nature.com/articles/s41434-020-0141-0.pdf
Reference43 articles.
1. Hagerman RJ, Berry-Kravis E, Hazlett HC, Bailey DB, Moine H, Kooy RF, et al. Fragile X syndrome. Nat Rev Dis Primers. 2017;3:17065.
2. Haenfler JM, Skariah G, Rodriguez CM, Monteiro da Rocha A, Parent JM, Smith GD, et al. Targeted reactivation of FMR1 transcription in fragile X syndrome embryonic stem cells. Front Mol Neurosci. 2018;11:1–17.
3. Maddalena A, Richards CS, McGinniss MJ, Brothman A, Desnick RJ, Grier RE, et al. Technical standards and guidelines for fragile X: the first of a series of disease-specific supplements to the standards and guidelines for clinical genetics laboratories of the American College of Medical Genetics. Genet Med. 2001;3:200–5.
4. Jiraanont P, Kumar M, Tang HT, Espinal G, Hagerman PJ, Hagerman RJ, et al. Size and methylation mosaicism in males with fragile X syndrome. Expert Rev Mol Diagn. 2017;17:1023–32.
5. Luo SY, Wu LQ, Duan RH. Molecular medicine of fragile X syndrome: based on known molecular mechanisms. World J Pediatr. 2016;12:19–27.
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