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, 导致广泛的临床表现. These disorders can range from mild to severe, and some can be life-threatening. 干细胞疗法 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
干细胞是未分化的细胞，具有分化为各种细胞类型的细胞, 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 (HSC): 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 (MSC): These cells are found in various tissues, 包括骨髓, 脂肪组织, 和脐带.
- 诱导多能干细胞 (ipscs): 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
造血干细胞移植 (HSCT) has been used for decades to treat severe inherited liver enzyme disorders. 在HSCT中, 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, 包括移植物与宿主病 (GVHD).