The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
May. 07, 2024
Filed:
Dec. 05, 2017
The Regents of the University of California, Oakland, CA (US);
Donald B. Kohn, Tarzana, CA (US);
Richard A. Morgan, Los Angeles, CA (US);
Roger P. Hollis, Los Angeles, CA (US);
THE REGENTS OF THE UNIVERSITY OF CALIFORNIA, Oakland, CA (US);
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 (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”.