Stem Cells in the Treatment of Inherited Liver Enzyme Disorders
Inherited liver enzyme disorders are a group of genetic conditions that affect the function of liver enzymes, leading to a wide range of clinical manifestations. These disorders can range from mild to severe, and some can be life-threatening. 干细胞治疗 has emerged as a promising approach for the treatment of inherited liver enzyme disorders, offering the potential for disease modification and even cure.
Pathophysiology of Inherited Liver Enzyme Disorders
Inherited liver enzyme disorders are caused by mutations in genes encoding enzymes involved in various metabolic pathways. These mutations can lead to the accumulation of toxic metabolites, which can damage liver cells and cause liver failure. The severity of the disorder depends on the specific enzyme deficiency and the extent of the metabolic imbalance.
Stem Cell Sources for Liver Disease Treatment
Stem cells are undifferentiated cells that have the potential to differentiate into various cell types, 包括肝细胞. Several sources of stem cells can be used for the treatment of liver disease, 包括:
- 造血干细胞 (造血干细胞): These cells are found in the bone marrow and can be collected through a procedure called bone marrow aspiration.
- 间充质干细胞 (间充质干细胞): 这些细胞存在于各种组织中, 包括骨髓, 脂肪组织, and umbilical cord.
- 诱导多能干细胞 (诱导多能干细胞): These cells are generated by reprogramming adult cells into a pluripotent state, which allows them to differentiate into any cell type.
造血干细胞移植
Hematopoietic stem cell transplantation (造血干细胞移植) has been used for decades to treat severe inherited liver enzyme disorders. In HSCT, HSCs from a healthy donor are transplanted into the patient, replacing the defective liver cells. HSCT can be curative for certain disorders, but it is associated with significant risks, including graft-versus-host disease (移植物抗宿主病).
