학술논문
In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions
Document Type
article
Author
Source
Nature Communications, Vol 13, Iss 1, Pp 1-15 (2022)
Subject
Language
English
ISSN
2041-1723
Abstract
Hereditary tyrosinemia type 1 (HT1) is an inborn error of metabolism caused by a deficiency in fumarylacetoacetate hydrolase (FAH). Here, the authors show in an animal model that HT1 can be treated via in vivo portal vein administration of a lentiviral vector carrying the human FAH transgene.