发明名称 GENE THERAPY VECTORS FOR ADRENOLEUKODYSTROPHY AND ADRENOMYELONEUROPATHY
摘要 The present invention provides compositions comprising retroviral vectors, transduced cells, and methods of using the same for gene therapy. In particular, the present invention relates to lentiviral vectors and cells transduced with those vectors to provide gene therapy to subjects having an adrenoleukodystrophy and/or adrenomyeloneuropathy.
申请公布号 KR20150029756(A) 申请公布日期 2015.03.18
申请号 KR20157004059 申请日期 2012.06.08
申请人 BLUEBIRD BIO, INC. 发明人 DENARO MARIA JOANN;FINER MITCHELL HOWARD;VERES GABOR
分类号 A61K48/00;C12N5/0735;C12N5/074;C12N5/0789;C12N5/10;C12N15/867 主分类号 A61K48/00
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