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publicationDate 2018-06-14-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber WO-2018106724-A1
titleOfInvention Optimized lentiviral vector for stem cell gene therapy of hemoglobinopathies
abstract 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 (βΑ83)). In certain embodiments the vectors described herein comprise novel defined LCR HS core sequences (HS2(~420 bp), HS3(~340 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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