Gene therapy for bleeding disorders.
Gene therapy for bleeding disorders.
复制标题
出血性疾病的基因治疗。
DOI：
10.1097/00062752-200211000-00008
复制
发表时间：
2002
影响因子：
3.2
通讯作者：
Manno,CatherineS
中科院分区：
文献类型：
--
作者：
Manno,CatherineS
The goal of gene therapy for bleeding disorders is to provide stable insertion and expression of a particular gene whose absence is responsible for a particular disease. The bleeding disorders for which the most basic and clinical research has been completed are the hemophilias factor VIII deficiency and factor IX deficiency. These X-linked diseases are caused by single-gene mutations; replacement of the defective gene has not only resulted in clinical and biochemical improvement in animal models but also provided promising results in phase I clinical trials. An ideal gene transfer approach to the treatment of hemophilia would require a minimally invasive procedure for gene delivery, have minimal associated morbidity, and result in long-term transgene expression, ideally yielding factor levels in the therapeutic range. Multiple approaches to gene transfer in the hemophilias are currently under investigation.
