Tyrosinemia Type 1 News and Research

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Penn Medicine and CHOP receive $26 million NIH grant to develop treatments for three rare genetic diseases

A Penn Medicine and Children's Hospital of Philadelphia (CHOP) team will seek to develop treatments for three rare, incurable genetic diseases with the help of a $26 million grant from the National Institutes of Health (NIH).

1 Aug 2023

Dietary induction of hepatocyte regeneration may be a viable strategy to enhance gene repair

Use of thyroid hormone to boost hepatocyte proliferation enhanced the efficiency of CRISPR/Cas9-mediated gene correction in the mouse liver.

11 Nov 2020

Researchers use CRISPR-Cas9 to prevent congenital disease in utero

Researchers have successfully used prenatal gene editing (CRISPR-Cas9) to prevent a fatal metabolic disorder in an animal model.

8 Oct 2018

Researchers test new approach to treat metabolic diseases without organ transplant

With a shortage of donor organs, Mayo Clinic is exploring therapeutic strategies for patients with debilitating liver diseases. Researchers are testing a new approach to correct metabolic disorders without a whole organ transplant. Their findings appear in Science Translational Medicine.

27 Jul 2016

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

University of Massachusetts Medical School researchers have found a way to more efficiently delivery a CRISPR/Cas9 therapeutic to adult mice with the metabolic disease Tyrosinemia type I that may also prove to be safer for use in humans.

2 Feb 2016