Durable correction of inherited metabolic liver disorders requires preventing transgene off-targeting from gene therapy vectors: the value of microRNAs.
Gonzalez-Aseguinolaza
Gloria
G
Prieto
Jesus
J
D000818:Animals; D000906:Antibodies / Q000097:blood; D000938:Antigen-Presenting Cells / Q000276:immunology; D001663:Bilirubin / Q000097:blood; D015415:Biomarkers / Q000097:blood; D003414:Crigler-Najjar Syndrome / Q000201:enzymology / Q000235:genetics / Q000276:immunology / Q000628:therapy*; D004195:Disease Models, Animal; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D014453:Glucuronosyltransferase / Q000096:biosynthesis / Q000235:genetics* / Q000276:immunology; D006801:Humans; D016086:Lentivirus / Q000235:genetics*; D008099:Liver / Q000201:enzymology*; D035683:MicroRNAs / Q000378:metabolism*; D020871:RNA Stability; D012333:RNA, Messenger / Q000378:metabolism; D051381:Rats; D011911:Rats, Gunn; D000099148:UGT1A1 Enzyme
D000906:Antibodies; D001663:Bilirubin; D015415:Biomarkers; D014453:Glucuronosyltransferase; D035683:MicroRNAs; D012333:RNA, Messenger; D000099148:UGT1A1 Enzyme