Primary hyperoxaluria, phenylketonuria, Gaucher disease, methylmalonic acidemia, propionic acidemia, isovaleric acidemia, type I glutaric acidemia, maple syrup urine disease, aminoaciduria — these are all examples of inherited rare metabolic diseases. These diseases comprise a diverse group of disorders with varying disease prevalence, and the prevalence of single disease is relatively low, but overall incidence rate is high, thus pose serious health risks. Cyagen has developed a comprehensive range of gene knockout, knock-in, and point mutation models for metabolic diseases. We also offer customized or collaborative development services tailored to meet the specific needs of researchers.
Disease Name | Target Genes | Targeting Methods | Ordering |
Aromatic L-amino acid decarboxylase deficiency, AADCD | Ddc | Mu | Order Now |
BH4-deficient hyperphenylalaninemia | Pts | KO | Order Now |
Biotinidase deficiency, BTDD | Btd | KO | Order Now |
Citrullinemia | Ass1 | KO | Order Now |
Fabry disease, FD | Gla | KO | Order Now |
Familial hypercholesterolemia, FH | Apoa2 | KO | Order Now |
Fish odour syndrome | Fmo3 | KO | Order Now |
Galactosemia | Galt | KO | Order Now |
Hereditary fructose intolerance, HFI | Aldob | KO | Order Now |
Hereditary tyrosinemia type 1, HT1 | Fah | KO | Order Now |
Hereditary tyrosinemia type 2, HT2 | Gcdh | KO | Order Now |
Hyperargininemia | Arg1 | KO | Order Now |
Inherited Hypomagnesemia, IH | Cldn16 | KO | Order Now |
Krabbe disease | Galc | Mu | Order Now |
Maple syrup urine disease, MSUD | Ppm1k | KO | Order Now |
Dbt | KO | Order Now | |
Medium-chain acyl-CoA dehydrogenase deficiency, MCADD | Acadm | KI | Order Now |
Methylmalonic acidemia, MMA | Mmut | KO | Order Now |
Ornithine transcarbamylase deficiency, OTCD | Otc | KO | Order Now |
Phenylketonuria, PKU | Pah | KO | Order Now |
Primary hyperoxaluria type 1, PH1 | Agxt | KO | Order Now |
Primary hyperoxaluria type 2, PH2 | Grhpr | KO | Order Now |
Sanfilippo syndrome | Naglu | KO | Order Now |