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:
Mar. 30, 2021
Filed:
Jul. 17, 2018
Csl Behring Gene Therapy, Inc., Pasadena, CA (US);
Csl Gene Therapy, Pty Ltd, Parkville, AU;
Jeffrey Ahlers, Winterville, NC (US);
Jeffrey Bartlett, Columbus, OH (US);
Chi-Lin Lee, Arcadia, CA (US);
Gene-Errol Eugenio Ringpis, West Hollywood, CA (US);
Geoffrey Phillip Symonds, Rose Bay, AU;
Ming Yan, Encino, CA (US);
CSL Behring Gene Therapy, Inc., Pasadena, CA (US);
CSL Gene Therapy Pty Ltd, Parkville, AU;
Abstract
The present disclosure provides expression vectors comprising at least two nucleic acid sequences, namely a nucleic acid sequence encoding an anti-HPRT RNAi, and a nucleic acid sequence encoding a gamma globin gene. In some embodiments, the viral vector is a self-inactivating lentiviral vector. In some embodiments, the gamma-globin gene is used to genetically correct sickle cell disease or β-thalassemia or to reduce symptoms thereof.