METHODS AND COMPOSITIONS FOR TREATMENT OF TUBEROUS SCLEROSIS COMPLEX

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专利权人:

Loon-Tzian Lo

发明人:

Loon-Tzian Lo

申请号：

US13390107

公开号：

US20120252877A1

申请日:

2010.08.16

申请国别(地区):

US

年份:

2012

代理人:

摘要：

The present disclosure relates to a method of treating Tuberous Sclerosis Complex (TSC). The present disclosure further provides an isolated polynucleotide molecule comprising, a polynucleotide comprising an AAV9 vector a polynucleotide encoding a TSC1 or a TSC2, or variant thereof and a promoter. The present disclosure further provides a pharmaceutical composition comprising an isolated polynucleotide molecule.

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