Search Results - john+tisdale

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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

Treatment of the beta-globinopathies through inhibition of RIOK3 activity

Disorders of adult beta-globin synthesis, which include sickle cell disease (SCD) and beta-thalassemia, are the most common monogenic disorders in the world. While the curative potential of bone marrow transplantation has been demonstrated, this approach is limited to a small fraction of affected patients due to the requirement for an HLA-matched donor,...

Published: 4/8/2024 | Inventor(s): John Tisdale, Bjorg Gudmundsdottir, Laxminath Tumburu

Keywords(s):

Category(s): Application > Therapeutics, Collaboration Sought > Licensing, TherapeuticArea > Immunology, 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: 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