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在新生儿及幼儿期,几乎所有被诊断为肝脏先天代谢异常性疾病的病例(表),在其肝脏功能障碍进展的同时都出现精神障碍。目前,对这类疾病的任何一种还没有根治性疗法。1980年人们利用环孢菌素A使全肝脏移植的成功率显著提高。1988年美国匹兹堡大学外科的Starzl等认为,这类疾病是肝脏移植的最好适应证,而对很多肝脏先天代谢异常性病人进行了肝移植手术。1988年报道,小儿达63例,成人达35例。但是,对此类疾病,即使不进行全肝移植,而仅补充缺损的酶,也是根本性的和理想的治疗方法。本文拟就以下两个问题进行探讨,其一,用肝细胞移植方法治疗肝脏先天代谢异常性疾病是否可行,目前的情况如何,还存在着什么问题。其二,把所缺损的基因植入肝细胞内进行治疗,以及在进行此种疗
In neonates and early childhood, almost all cases diagnosed with idiopathic metabolic abnormalities of the liver (tables) develop mental disorders at the same time as their liver dysfunction progresses. At present, there is no radical treatment for any of these diseases. In 1980, people using cyclosporin A significantly increased the success rate of total liver transplantation. Starzl et al., Department of Surgery, University of Pittsburgh, 1988, concluded that these diseases are the best indications for liver transplantation and that liver transplantation is performed on many patients with idiopathic metabolic abnormalities of the liver. Reported in 1988, 63 cases of pediatric, adult 35 cases. However, it is a fundamental and ideal treatment for such diseases, even if the whole liver transplantation is not carried out, and only the deficient enzymes are added. This article intends to explore the following two issues, first, the use of liver cell transplantation for the treatment of liver anomalies is an acceptable natural disease, the current situation, what are the remaining problems. Second, the defect of the gene implanted in liver cells for treatment, as well as in the treatment