换一批Oligonucleotides Hold Promise as a Therapy for Friedreich's Ataxia: Friedreich's ataxia currently is incurable, but synthetic antisense oligonucleotides have demonstrated promising results in increasing frataxin gene expression and restoring it to normal levels.
主题词
弗里德赖希共济失调(Friedreich Ataxia);人类(Humans);铁结合蛋白质类(Iron-Binding Proteins);寡核苷酸类(Oligonucleotides);寡核苷酸类, 反义(Oligonucleotides, Antisense)
DOI
10.1002/ajmg.a.38850
PMID
29878710
发布时间
2023-12-13
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