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Treatment of Cystathionine β-Synthase Deficiency in Mice Using a Minicircle-Based Naked DNA Vector.

Hum Gene Ther. 2019; 
Lee Hyung-Ok,Gallego-Villar Lorena,Grisch-Chan Hiu Man,Häberle Johannes,Thöny Beat,Kruger Warr
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摘要

Cystathionine β-synthase () deficiency is a recessive inborn error of metabolism characterized by extremely elevated total homocysteine (tHcy) in the blood. Patients diagnosed with CBS deficiency have a variety of clinical problems, including dislocated lenses, osteoporosis, cognitive and behavioral issues, and a significantly increased risk of thrombosis. Current treatment strategies involve a combination of vitamin supplementation and restriction of foods containing the homocysteine precursor methionine. Here, a mouse model for CBS deficiency () was used to evaluate the potential of minicircle-based naked DNA gene therapy to treat CBS deficiency. A 2.3 kb DNA-minicircle containing the liver-speci... More

关键词

amino acid metabolism,homocystinuria,hydrodynamic injection,inborn error,minicircles,non-viral gene the