Deletion of the GAA repeats from the human frataxin gene using the CRISPR-Cas9 system in YG8R-derived cells and mouse models of Friedreich ataxia
Author:
Publisher
Springer Science and Business Media LLC
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
https://www.nature.com/articles/gt201689.pdf
Reference53 articles.
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3. Harding AE . Friedreich's ataxia: a clinical and genetic study of 90 families with an analysis of early diagnostic criteria and intrafamilial clustering of clinical features. Brain 1981; 104: 589–620.
4. Lynch DR, Farmer JM, Balcer LJ, Wilson RB . Friedreich ataxia: effects of genetic understanding on clinical evaluation and therapy. Arch Neurol 2002; 59: 743–747.
5. Pandolfo M . Molecular pathogenesis of Friedreich ataxia. Arch Neurol 1999; 56: 1201–1208.
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