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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

2021-052 BOOST CARDIAC REPROGRAMMING BY MODULATING IMMUNE CELLS AND MICROENVIRONMENT

SUMMARYResearchers from the Department of Bioengineering at UCLA have identified key players in in both preventing and boosting cardiac reprogramming. This innovation can be used for in vivo human cardiac reprogramming to provide a novel approach for cardiac regeneration in the clinic and other fields of regenerative medicine. BACKGROUNDIschemic heart...

Published: 9/5/2023 | Inventor(s): Song Li

Keywords(s):

Category(s): Platforms > Drug Delivery, Platforms > Drug Delivery > Peptide Base, Platforms > Drug Delivery > Viral Delivery Systems, Therapeutics, Therapeutics > Cardiovascular

2015-401 SELF-AMPLIFYING NON-CODING RNA FOR TARGETED GENE KNOCKDOWN

UCLA researchers have developed a novel delivery platform for efficient and specific gene silencing in RNAi therapy. BACKGROUND:RNA interference (RNAi) and microRNA are potent methods for efficient and specific gene silencing. The technologies have been extensively exploited to develop therapies for treating fatal disorders like cancer and neurogenetic...

Published: 7/19/2023 | Inventor(s): William Gelbart, Charles Knobler, Feng Guo

Keywords(s): Drug Delivery, Viral Delivery Systems

Category(s): Platforms > Drug Delivery > Viral Delivery Systems

Biotinylated Ligand-Directed Targeting Lentiviral Vectors

SummaryUCLA researchers in the Department of Medicine have developed a novel method to conjugate targeting ligands on lentiviral vectors. The method allows for selective transduction of mammalian cells types avoiding non-target organs.BackgroundGene therapy is a promising method to treat genetic disorders. Lentiviral vectors are used as a gene transduction...

Published: 7/19/2023 | Inventor(s):

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

Category(s): Life Science Research Tools, 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