Csl Behring Gene Therapy, Inc.

Csl Gene Therapy Pty Ltd.

Jeffrey Ahlers

Geoffrey Phillip Symonds

Ming Yan

Jeffrey S Bartlett

Chi-Lin Lee

Gene-Errol Eugenio Ringpis

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 &#x3b2;-thalassemia or to reduce symptoms thereof.

Growing community of inventors

Winterville, NC, United States of America

Jeffrey Ahlers