Correction/mutation of acid α-D-glucosidase gene by modified single-stranded oligonucleotides: in vitro and in vivo studies
Author:
Publisher
Springer Science and Business Media LLC
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
http://www.nature.com/articles/3302096.pdf
Reference27 articles.
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2. Hers HG . Alpha-glucosidase deficiency in generalized glycogen storage disease. Biochem J 1963; 86: 11–16.
3. Loonen MC et al. Identification of heterozyotes for glycogenosis 2. Clin Genet 1981; 19: 55–63.
4. Gillette PC, Nihill MR, Singer DB . Electrophysiological mechanism of the short PR interval in Pompe disease. Am J Dis Child 1974; 12: 622–626.
5. Metzl JD, Elias ER, Berul CI . An interesting case of infant sudden death: severe hypertrophic cardiomyopathy in Pompe's disease. Pacing Clin Electrophysiol 1999; 22: 821–822.
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