Search Results - naoya+uchida

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Cas9 Protein Delivery with Lentiviral Vector Particles as a Therapy for Sickle Cell Disease
This technology includes efficient lentiviral gene delivery system for both guide RNA and Cas9 endonuclease as a method to cure sickle cell disease. Gene correction is an ideal gene therapy strategy for hereditary disease, including sickle cell disease. To deliver both guide RNA and Cas9 endonuclease into target cells, we used HIV-l based lentiviral...
Published: 8/12/2024   |   Inventor(s): Naoya Uchida, Juan Haro Mora, John Tisdale
Keywords(s):  
Category(s): Application > Therapeutics, Collaboration Sought > Licensing, ResearchProducts > Plasmids/Vectors
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: 10/28/2024   |   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