OPTIMIZED LENTIVIRAL VECTOR FOR STEM CELL GENE THERAPY OF HEMOGLOBINOPATHIES

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专利权人:

The Regents of the University of California

发明人:

Donald B. Kohn,Richard A. Morgan,Roger P. Hollis

申请号：

US16466970

公开号：

US20200109416A1

申请日:

2017.12.05

申请国别(地区):

US

年份:

2020

代理人:

摘要：

In certain embodiments an optimized derivative of the CCLc-βAS3-FB lentiviral vector termed (CCLc-mGata/ANK-CoreLCR-βAS3-FB), is provided which is capable of driving lineage-restricted expression of a beta-globin gene (e.g., an anti-sickling β-globin like gene (βA83)). In certain embodiments the vectors described herein comprise novel defined LCR HS core sequences (HS2(˜420 bp), HS3β40 bp), HS4(˜410 bp)) which can be used to replace the putative LCR HS sequences present within the “mini-LCR” (˜3.6 kb reduced to ˜1.2 kb) to produce an “optimized mini-LCR”.

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