Chapter title |
Fah Knockout Animals as Models for Therapeutic Liver Repopulation
|
---|---|
Chapter number | 20 |
Book title |
Hereditary Tyrosinemia
|
Published in |
Advances in experimental medicine and biology, January 2017
|
DOI | 10.1007/978-3-319-55780-9_20 |
Pubmed ID | |
Book ISBNs |
978-3-31-955779-3, 978-3-31-955780-9
|
Authors |
Markus Grompe |
Abstract |
Several animal models of Fah deficiency have been developed, including mice, pigs and most recently rats. Initially, the murine models were developed with the intent to mirror the human disease for pathophysiologic and therapeutic studies. However, it soon became apparent that Fah-positive hepatocytes have a potent selective growth advantage in mutant liver and can extensively repopulate the diseased organ. For this reason, Fah mutant mice have become a workhorse for liver biology and are widely used in liver stem cell and hepatic gene therapy research. Immune deficient Fah-knockout mice can be repopulated with human hepatocytes, creating "mice with human livers". These chimeric animals have become an important preclinical model for infectious diseases, metabolism and gene therapy. The potent expansion of human hepatocytes in Fah knockout mice has given rise to the concept of using Fah mutants as living bioreactors to produce large quantities of fully mature hepatocytes. As a consequence, larger animal models of Fah deficiency have recently been developed. |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 39 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Ph. D. Student | 15 | 38% |
Researcher | 7 | 18% |
Student > Postgraduate | 4 | 10% |
Student > Doctoral Student | 2 | 5% |
Professor | 1 | 3% |
Other | 3 | 8% |
Unknown | 7 | 18% |
Readers by discipline | Count | As % |
---|---|---|
Biochemistry, Genetics and Molecular Biology | 13 | 33% |
Medicine and Dentistry | 7 | 18% |
Pharmacology, Toxicology and Pharmaceutical Science | 4 | 10% |
Agricultural and Biological Sciences | 3 | 8% |
Chemical Engineering | 1 | 3% |
Other | 2 | 5% |
Unknown | 9 | 23% |