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A Novel Therapeutic Vector for Hemoglobin Disorders

Investigators at the National Heart, Lung, and Blood Institute have designed a novel lentiviral vector as a potential gene therapy for sickle cell anemia and beta-thalassemia. The novel lentiviral vector encodes the beta-globin gene in a forward orientation and can produce 5-10 fold higher viral titer and 4-10 fold higher gene transfer efficiency to...

Published: 7/25/2024 | Inventor(s): John Tisdale, Naoya Uchida

Keywords(s): Development, DISORDERS, Forward-oriented, Generation, hemoglobin, Listed LPM Maddox as of 4/15/2015, Post LPM Assignment Set 20150420, Pre LPM working set 20150418, therapeutic, Therpeutic, Vector, VPXXXX, WJXXXX, XEXXXX, YAXXXX, YBXXXX, YCXXXX

Category(s): Collaboration Sought > Collaboration, Application > Therapeutics, TherapeuticArea > Endocrinology, TherapeuticArea > Dental, TherapeuticArea > Ophthalmology, TherapeuticArea > Cardiology, TherapeuticArea > Infectious Disease, TherapeuticArea > Oncology