使用基于微环的裸 DNA 载体治疗小鼠中的胱硫氨酸 β - 合成酶缺乏症。
关键词: amino acid metabolism homocystinuria hydrodynamic injection inborn error minicircles non-viral gene therapy
Mesh : Animals Biomarkers Cystathionine beta-Synthase / blood deficiency genetics DNA, Circular / administration & dosage genetics Disease Models, Animal Female Gene Expression Gene Transfer Techniques Genetic Therapy Genetic Vectors / administration & dosage genetics Homocystinuria / genetics metabolism therapy Liver / metabolism pathology Male Mice Mice, Knockout Phenotype Transfection / methods Treatment Outcome
来 源:
DOI:10.1089/hum.2019.014
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