Search Results - roger+hollis

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Gene Editing of Monogenic Disorders in Human Hematopoietic Stem Cells

Case 2018-372Researchers at the UCLA Department of Microbiology, Immunology & Molecular Genetics have developed novel methods to achieve efficient, precise gene integration and effective expression of cDNA cassettes to express normal versions of genes in hematopoietic stem cells.BACKGROUNDHematopoietic stem cells (HSCs) have great therapeutic potential...

Published: 6/3/2026 | Inventor(s): Donald Kohn, Caroline Kuo, Zulema Romero Garcia, Roger Hollis

Keywords(s): Drug Delivery, Gene Editing Systems, Gene Therapy, Life Science Research Tools

Category(s): Platforms > Drug Delivery, Therapeutics > Gene Therapy And Editing, Life Science Research Tools

Augmentations to Lentiviral Vectors to Increase Expression (UCLA Case No. 2019-616)

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 cell disease (SCD) is one of the...

Published: 7/17/2025 | Inventor(s): Donald Kohn, Roger Hollis, Richard Morgan

Keywords(s):

Category(s): Therapeutics > Gene Therapy And Editing, Life Science Research Tools, Therapeutics > Genetic Diseases

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: 7/17/2025 | 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: 7/17/2025 | 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