The future of gene-targeted therapy for hereditary tyrosinemia type 1 as a lead indication among the inborn errors of metabolism.

Whitney S ThompsonGourish MondalCaitlin J Vanlith Robert A Kaiser Joseph B Lillegard

Published in: Expert opinion on orphan drugs (2020)

It is proposed that not only is HT1 a safe indication for gene therapy, its unique characteristics position it to be an ideal IEM to develop for clinical investigation.

Keyphrases

gene therapy
current status
copy number
emergency department
genome wide identification
genome wide analysis
drug induced