Progress in Gene Therapy for Hereditary Tyrosinemia Type 1

Abstract excerpt

Hereditary Tyrosinemia Type-1 (HT1), an inherited error of metabolism caused by a mutation in the fumarylacetoacetate hydrolase gene, is associated with liver disease, severe morbidity, and early mortality. The use of NTBC (2-(2-nitro-4-fluoromethylbenzoyl)-1,3-cyclohexanedione) has almost eradicated the acute HT1 symptoms and childhood mortality. However, patient outcomes remain unsatisfactory due to the...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to CRISPR and Genetic Engineering
• Share to Viral gastroenteritis research and epidemiology
• Share to Virus-based gene therapy research