Substrate deprivation therapy for MPS disorders

本提案的目的是评估一种新的治疗方案，以儿童遗传性疾病称为粘多糖病（MPS）。由于缺乏降解所需的酶，多磺酸粘多糖产生于体内细胞中复杂碳水化合物的积累。通过减少碳水化合物的合成，我们可以控制储存碳水化合物的水平，减轻与MPS相关的病理。这种新疗法是基于抑制碳水化合物合成的葡萄糖的化学修饰，被称为底物剥夺疗法。

The aim of this proposal is to evaluate a novel therapy option for children with a genetic disorder called mucopolysaccharidosis (MPS). MPS arise from the build up of complex carbohydrates in cells within the body due to the deficiency of an enzyme required for their degradation. By decreasing the synthesis of carbohydrate we can manipulate the level of stored carbohydrate and alleviate the pathology associated with MPS. The novel therapy is based on a chemical modification of glucose that inhibits carbohydrate synthesis and is termed substrate deprivation therapy.