Advanced searches left 3/3

Tyrosinemia - BioProject

Summarized by Plex Scholar
Last Updated: 06 February 2022

* If you want to update the article please login/register

In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions

In the porcine model of HT1, we show for the first time a cure of HT1 by direct injection of a therapeutic lentiviral vector aimed at the expression of a human fumarylacetoacetate hydrolase transgene. Indeed, the phenotypic and genomic results from the in vivo lentiviral vector research show comparative advantage over other therapies, including ex vivo cell therapy, and thus encourage clinical application of this strategy. Comparison of animal RNA expression profiles of WT animals treated with HT1: NTBC, LV-FAH gene therapy, and the negative control, which was cycled on and off the level of care NTBC was included in the WT's standard of care NTBC. LV-FAH from various organs in the LV-FAH treated animals was also evaluated for integration profiles.

Source link: https://www.ncbi.nlm.nih.gov/bioproject/781617

* Please keep in mind that all text is summarized by machine, we do not bear any responsibility, and you should always check original source before taking any actions

* Please keep in mind that all text is summarized by machine, we do not bear any responsibility, and you should always check original source before taking any actions