ENZYME REPLACEMENT THERAPY IN DISORDERS THAT AFFECT THE CENTRAL NERVOUS SYSTEM

影响中枢神经系统疾病的酶替代疗法

• 批准号: 2579614
• 负责人: R O BRADY
• 金额: --
• 依托单位: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/2579614
• 关键词: Gaucher's disease; Mammalia; beta galactosidase; brain; central nervous system; drug delivery systems; drug metabolism; drug receptors; enzyme therapy; gangliosides; gangliosidosis GM1; glucosylceramidase; inborn metabolism disorder; injection /infusion; metabolism disorder chemotherapy; neurons; nonhuman therapy evaluation; placenta; tissue /cell culture

Enzyme replacement therapy has been shown to be extraordinarily effective for patients with Type 1 (non-neuronopathic) Gaucher's disease. We are now developing procedures to deliver useful amounts of enzymes to the brain in patients with hereditary metabolic storage disorders. We have examined the effect of human placental beta-galactosidase on the amount of ganglioside GM1 in animal analogues of human generalized (GM1) gangliosidosis using a new intracerebral protein delivery system. We are also determining the distribution of glucocerebrosidase in the brain using convection-enhanced intracerebral injection of this enzyme. We are characterizing the receptors involved in the uptake of glucocerebrosidase and other enzymes by neurons in tissue culture.

酶替代疗法已被证明非常有效