Title : AAV8 Gene Therapy Rescues the Newborn Phenotype of a Mouse Model of Crigler-Najjar.

Pub. Date : 2018 Jul

PMID : 29448836






3 Functional Relationships(s)
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1 Newborn UGT1 KO mice treated with AAV had serum total bilirubin levels that were 5.7 times higher than the levels seen in heterozygous and wild-type mice, likely due to dilution of vector genome copies (GC) in the liver resulting from a proliferation of hepatocytes during growth of the animal. Bilirubin UDP glucuronosyltransferase 1 family, polypeptide A2 Mus musculus
2 The elevation in serum total bilirubin levels in adult UGT1 KO mice depended on the AAV8 vector dose. Bilirubin UDP glucuronosyltransferase 1 family, polypeptide A2 Mus musculus
3 Therefore, a low-level and likely transient decrease in serum total bilirubin during the first days of life is necessary for rescuing the lethal phenotype present in the neonatal UGT1 KO mouse. Bilirubin UDP glucuronosyltransferase 1 family, polypeptide A2 Mus musculus