Blood into Liver: Miracle, Bio Revolution, Clinical Promise―or All Three?

摘要

Science Watch found it hard to decide if paper #5 should be allocated to medicine or biology. The work was supported by the U.S. National Institutes of Health and the American Liver Foundation; it attracted an accompanying commentary by a children's cancer specialist; and it rode on the medical horse in Nature's stable. However, the research is in mice―in particular, mice rendered deficient in the enzyme fumarylacetoacetate hy-drolase (FAH), which constitute an animal model for a human inborn error of metabolism called tyrosinemia type Ⅰ. Inherited enzyme deficiencies generally are not easy to treat, and clinical management has included drugs, dietary control, enzyme replacement, organ transplantation, and gene therapy In tyrosinemia, with its life-threatening liver damage, there has been some clinical success (and in the murine model too) with the compound 2-(2-nitro-4-trifluoromethylbenzyol)-1,3-cyclohex-anedione, but a novel approach is offered by the dawning realization that stem cells may demonstrate plasticity―in short, neural stem cells are not committed only to the nervous system, for example, while hematopoietic stem cells may give rise to liver and muscle cells as well as the expected blood cells of various types.