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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

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Category(s): Application > Therapeutics, Collaboration Sought > Licensing, ResearchProducts > Plasmids/Vectors