intrathecal delivery of abcd1-encoding nucleic acid sequences for the treatment of adrenomyeloneuropathy

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专利权人:

THE GENERAL HOSPITAL CORPORATION

发明人:

CASEY A. MAGUIRE,FLORIAN EICHLER

申请号：

BR112018008993

公开号：

BR112018008993A2

申请日:

2016.11.03

申请国别(地区):

BR

年份:

2018

代理人:

摘要：

The present invention relates to methods encompassing the delivery of abcd1 nucleic acid sequences for the treatment of x-linked adrenoleukodystrophy (x-ald), for example, for adrenomyeloneuropathy (amn).a presente invenção refere-se a métodos que abrangem a entrega de sequências de ácidos nucleicos abcd1 para o tratamento de adrenoleucodistrofia ligada ao x (x-ald), por exemplo, para adrenomieloneuropatia (amn).

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/