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Optimized Lentiviral Vector for Stem Cell Gene Therapy of Hemoglobinopathies

UCLA Case No. 2017-460 Summary UCLA researchers in the Department of Microbiology, Immunology and Molecular Genetics have developed a novel method to produce short lentiviral vectors with tissue-specific expression, with a primary focus on lentiviral vectors for treating sickle cell disease and other disorders of hemoglobin. Background Sickle...

Published: 1/23/2024 | Inventor(s): Donald Kohn, Roger Hollis, Richard Morgan

Keywords(s): Drug Delivery, Gene Editing Systems, Gene Therapy, Genetic Disease, Life Science Research Tools, Plasmids / Vectors, Research Methods, Therapeutics & Vaccines, Viral Delivery Systems

Category(s): Life Science Research Tools > Plasmids/Vectors, Life Science Research Tools, Life Science Research Tools > Research Methods, Platforms > Drug Delivery, Therapeutics, Platforms > Drug Delivery > Viral Delivery Systems, Therapeutics > Gene Therapy And Editing

2018-862 GENERATION OF MINIMAL ENHANCER ELEMENTS USING MASSIVELY PARALLEL REPORTER ASSAYS

SummaryUCLA researchers in the Department of Microbiology, Immunology and Molecular Genetics have developed a novel method to produce short lentiviral vectors with tissue-specific expression, with a primary focus on lentiviral vectors for treating sickle cell disease and other disorders of hemoglobin.BackgroundSickle cell disease (SCD) is one of the...

Published: 7/19/2023 | Inventor(s): Donald Kohn, Roger Hollis, Richard Morgan, Aaron Cooper

Keywords(s): Drug Delivery, Gene Editing Systems, Gene Therapy, Genetic Disease, Life Science Research Tools, Plasmids / Vectors, Research Methods, Therapeutics & Vaccines, Viral Delivery Systems

Category(s): Life Science Research Tools, Life Science Research Tools > Plasmids/Vectors, Life Science Research Tools > Research Methods, Platforms > Drug Delivery, Therapeutics, Platforms > Drug Delivery > Viral Delivery Systems, Therapeutics > Gene Therapy And Editing