Tyrosinemia News and Research

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Tyrosinemia is a genetic disorder characterized by elevated blood levels of the amino acid tyrosine, a building block of most proteins. Tyrosinemia is caused by the shortage (deficiency) of one of the enzymes required for the multistep process that breaks down tyrosine. If untreated, tyrosine and its byproducts build up in tissues and organs, which leads to serious medical problems.
Drug increases melanin production in some people with albinism

Drug increases melanin production in some people with albinism

Yeast model may pave way for development of novel therapies for metabolic disorders

Yeast model may pave way for development of novel therapies for metabolic disorders

Researchers use CRISPR-Cas9 to prevent congenital disease in utero

Researchers use CRISPR-Cas9 to prevent congenital disease in utero

Nanoparticles carrying CRISPR gene editing tools for genetic modifications

Nanoparticles carrying CRISPR gene editing tools for genetic modifications

Researchers test new approach to treat metabolic diseases without organ transplant

Researchers test new approach to treat metabolic diseases without organ transplant

New way to more efficiently deliver CRISPR/Cas9 therapeutic to mice with Tyrosinemia type I

New way to more efficiently deliver CRISPR/Cas9 therapeutic to mice with Tyrosinemia type I

New gene-editing system holds potential for treating many genetic disorders

New gene-editing system holds potential for treating many genetic disorders

Nitisinone treatment effectively prevents tyrosinemia in children

Nitisinone treatment effectively prevents tyrosinemia in children

First demonstration of newly developed dual humanized FRGTM KO system against malaria

First demonstration of newly developed dual humanized FRGTM KO system against malaria

UCB, Synosia Therapeutics announce new strategic partnership in neurology

UCB, Synosia Therapeutics announce new strategic partnership in neurology

FDA clears blood test to screen newborn infants for a variety of inherited diseases

FDA clears blood test to screen newborn infants for a variety of inherited diseases