Large animal models and new therapies for glycogen storage disease.
Large animal models and new therapies for glycogen storage disease.
复制标题
DOI：
10.1007/s10545-014-9766-8
复制
发表时间：
2015-05
影响因子：
4.2
通讯作者：
Koeberl DD
中科院分区：
文献类型：
--
作者：
Brooks ED;Koeberl DD
Glycogen storage diseases (GSD), a unique category of inherited metabolic disorders, were first described early in the 20th century. Since then, the biochemical and genetic bases of these disorders have been determined, and an increasing number of animal models for GSD have become available. At least 7 large mammalian models have been developed for laboratory research on GSDs. These models have facilitated the development of new therapies, including gene therapy, which are undergoing clinical translation. For example, gene therapy prolonged survival and prevented hypoglycemia during fasting for greater than one year in dogs with GSD type Ia, and the need for periodic re-administration to maintain efficacy was demonstrated in that dog model. The further development of gene therapy could provide curative therapy for patients with GSD and other inherited metabolic disorders.
