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发明申请
US20090202515A1 AGENTS FOR SUPPRESSING NEURAL FIBROTIC DEGENERATION 审中-公开
用于抑制神经纤维变性的药剂

AGENTS FOR SUPPRESSING NEURAL FIBROTIC DEGENERATION
摘要：
The present invention examined the accumulation of chondroitin sulfate proteoglycans (CSPGs). The present invention relates to neurodegeneration-suppressing agents that are suitable for gene therapy or prevention of neural fibrotic degenerative diseases which induce neural cell death due to an accumulation of abnormal proteins, where the therapies are based on siRNAs against N-acetylgalactosamine-4-O-sulfotransferases (N-acetylgalactosamine-4-O-sulfotransferase-1, N-acetylgalactosamine-4-O-sulfotransferase-2, and N-acetylgalactosamine-4-sulfate 6-O-sulfotransferase (GalNAc4ST-1, GalNAc4ST-2, and GALNAC4S-6ST, respectively)), which are sulfotransferases for acetylgalactosamine, a CSPG side chain, and chondroitinase ABC, an enzyme that degrades chondroitin sulfate, another CSPG side chain.
摘要（中）：

本发明检测了硫酸软骨素蛋白聚糖（CSPG）的积累。 本发明涉及适用于基因治疗或预防由于异常蛋白质积累引起神经细胞死亡的神经纤维变性疾病的神经变性抑制剂，其中所述疗法基于针对N-乙酰半乳糖胺-4- O的siRNA - 转氨酶（N-乙酰半乳糖胺-4- O - 磺基转移酶-1，N-乙酰半乳糖胺-4- -O-磺基转移酶-2和N-乙酰半乳糖胺-4-硫酸酯6-O-磺基转移酶（GalNAc4ST-1，GalNAc4ST-2和 分别为GALNAC4S-6ST）），其为乙酰半乳糖胺磺酸转移酶，CSPG侧链和软骨素酶ABC，其是降解硫酸软骨素，另一CSPG侧链的酶。

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