了解琥珀酸半醛脱氢酶缺乏症 ( SSADHD ) 的分子机制 : 迈向 SSADH 靶向药物的发展。
关键词: ALDH5A1 GABA receptors enzyme replacement therapy epilepsy gene therapy inhibition mouse model plasticity succinic semialdehyde dehydrogenase deficiency (SSADHD) γ-aminobutyric acid (GABA) γ-hydroxybutyrate (GHB)
Mesh : Amino Acid Metabolism, Inborn Errors / metabolism Animals Child Developmental Disabilities / genetics Humans Mice Succinate-Semialdehyde Dehydrogenase / deficiency metabolism gamma-Aminobutyric Acid / metabolism
来 源:
DOI:10.3390/ijms23052606
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