Molecules able to modulate the expression of at least a gene involved in degradative pathways and uses thereof

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专利权人:

FONDAZIONE TELETHON

发明人:

SARDIELLO MARCO,BALLABIO ANDREA

申请号：

NZ61163610

公开号：

NZ611636A

申请日:

2010.02.11

申请国别(地区):

NZ

年份:

2015

代理人:

摘要：

Disclosed is a use of a TFEB protein having an amino acid sequence that consists of residues 1-476 of Seq Id No. 228 in the preparation of a medicament for the treatment of a neurodegenerative/lysosomal storage disorder wherein the sequence is as defined in the specification.

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