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Literature DB >> 30681273

Gene Editing Successfully Corrects 2 Amino Acid Disorders: In 2 preclinical studies using CRISPR-mediated gene editing, phenylketonuria and hereditary tyrosinemia type 1 were corrected.

Abstract

Entities: Disease

Mesh：

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Year: 2019 PMID： 30681273 DOI： 10.1002/ajmg.a.61026

Source DB: PubMed Journal: Am J Med Genet A ISSN： 1552-4825 Impact factor: 2.802

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1 in total

1. Repeated and progressive rhabdomyolysis due to a novel carnitine palmitoyltransferase II gene variant in an adult male: A case report.

Authors: Lina Shao; Chunya Liu; Liyuan Xu; Rizhen Yu; Yiwen Li; Maosheng Chen; Qiang He
Journal: Medicine (Baltimore) Date: 2019-11 Impact factor: 1.817

1 in total