Search Results - gene+editing+systems

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CHST6 Mini Gene - CHST6 Expression Construct With Native Promoter (UCLA Case No. 2023-301)

UCLA researchers from the Department of Human Genetics and Department of Ophthalmology have developed a mini gene encoding a protein which is mutated in macular corneal dystrophy, leading to a potential gene therapy for this disorder. BACKGROUND: Macular corneal dystrophy (MCD) is a rare genetic disease caused by mutations in the CHST6 gene, which...

Published: 11/20/2024 | Inventor(s): Wenlin Zhang, Anthony Aldave

Keywords(s): AAV, Adeno-associated viruses (AAV), Gene Editing Systems, Gene Therapy, genetics, keratinocytes, macular corneal dystrophy, mini gene, mutation, native promoter, Ophthalmology

Category(s): Therapeutics > Ophthalmology, Therapeutics > Gene Therapy And Editing

Methods and Compositions for Modification of the HPRT Locus (UCLA Case No. 2013-570)

UCLA researchers have developed a gene editing strategy and methodology based on nuclease-based insertion. BACKGROUND: Gene therapies are the forefront of therapeutic advancements for debilitating diseases. However, delivery and insertion of the therapeutic transgene are major hurdles that must be solved to effectively implement this technology....

Published: 4/12/2024 | Inventor(s): Noriyuki Kasahara

Keywords(s): Gene delivery, Gene Editing Systems, Gene Therapy

Category(s): Therapeutics > Gene Therapy And Editing

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

2021-056 CRISPR RIBONUCLEOPROTEIN COMPLEX GENOME EDITING OF HUMAN INNATE IMMUNE CELLS

Researchers in the Department of Microbiology, Immunology, and Molecular Genetics at UCLA have developed an optimized strategy for non-viral CRISPR-Cas9 ribonucleoprotein gene editing of primary innate immune cells. BACKGROUND:CRISPR (clustered, regularly interspaced, short palindromic repeats) genome engineering has become a powerful tool to investigate...

Published: 9/7/2023 | Inventor(s): Timothy O'Sullivan, Luke Riggan, Andrew Hildreth

Keywords(s): CRISPR, Gene Editing Systems

Category(s): Therapeutics > Gene Therapy And Editing

2019-880 ZIKA VIRUS-BASED ONCOLYTIC THERAPY FOR BRAIN CANCER

UCLA researchers in the Department of Molecular and Medical Pharmacology have developed a novel zika virus-based oncolytic therapy to treat brain cancer.BACKGROUND: Glioma is a type of brain tumor that accounts for 80% of all primary malignant brain tumors. Standard therapy for high-grade glioma requires a combination of surgical resection, radiation...

Published: 9/12/2023 | Inventor(s): Vaithilingaraja Arumugaswami, David Nathanson

Keywords(s): Cns, Drug Delivery, Gene Editing Systems, Gene Therapy, Immunology, Immunotherapy, Oncology, Oncology Immunotherapy, Plasmids / Vectors, Therapeutics & Vaccines, Viral Delivery Systems

Category(s): Diagnostic Markers > Immunology, Therapeutics > CNS and Neurology, Therapeutics > Vaccines, Therapeutics > Oncology, Therapeutics > Gene Therapy And Editing, Therapeutics > Oncology > Oncology Immunotherapy

2019-870 GENE EDITING OF MONOGENIC DISORDERS IN HUMAN HEMATOPOIETIC STEM CELLS (XHIM)

Researchers 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. BACKGROUND:Hematopoietic stem cells (HSCs)—stem cells that give rise to...

Published: 11/15/2024 | Inventor(s): Donald Kohn

Keywords(s): Gene Editing Systems, Gene Therapy, Genetic Disease, Hematology, Immunotherapy, iPS/stem cells, Therapeutics & Vaccines, Viral Delivery Systems

Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Hematology, Therapeutics > Immunology And Immunotherapy, Therapeutics > Stem Cells And Regenerative Medicine, Therapeutics > Stem Cells And Regenerative Medicine > Ips Stem Cells

2017-657: Gene Editing Stem Cells for Generating Non-Alloreactive T Cells for Immunotherapy

SUMMARY UCLA researchers in the Department of Pathology and Medicine have developed human stem cells that can be used to generate non-alloreactive, antigen specific T cells for adoptive cell therapy. BACKGROUND Adoptive cell therapy (ACT) with engineered T cells has shown unprecedented potential in the treatment of cancer, infectious diseases,...

Published: 9/5/2023 | Inventor(s): Gay Crooks, Christopher Seet, Amelie Montel Hagen

Keywords(s): Gene Editing Systems, Gene Therapy, iPS/stem cells, Life Science Research Tools, Therapeutics & Vaccines

Category(s): Life Science Research Tools, Therapeutics, Therapeutics > Gene Therapy And Editing, Therapeutics > Stem Cells And Regenerative Medicine > Ips Stem Cells

Gene Modified Autologous Dendritic Cells in Combination with Anti-PD1 Therapy for Metastatic Lung Cancer (UCLA Case No. 2019-844)

UCLA researchers at David Geffen School of Medicine have developed a novel approach of in situ vaccination by intratumorally injecting chemokine gene-modified dendritic cells to sensitize non-responsive non-small cell lung cancer (NSCLC) to anti-PD-1/PD-L1 immunotherapy. BACKGROUND:The treatment of non-small cell lung cancer (NSCLC) has...

Published: 4/9/2024 | Inventor(s): Steven Dubinett

Keywords(s): Drug Delivery, Gene Editing Systems, Gene Therapy, Immunotherapy, Medical Devices and Materials, Oncology, Oncology Immunotherapy, Therapeutics & Vaccines

Category(s): Therapeutics > Gene Therapy And Editing, Therapeutics > Oncology, Therapeutics > Immunology And Immunotherapy

2020-166 CHIMERIC ANTIGEN RECEPTORS AGAINST HUMAN CYTOMEGALOVIRUS

UCLA researchers from the Departments of Medicine and Infectious Diseases have developed novel chimeric antigen receptors against human cytomegalovirus. BACKGROUND:Human cytomegalovirus (CMV) is an infectious virus that is found in over half the world’s population. In most healthy people, the virus is suppressed by the body’s immune system....

Published: 11/15/2024 | Inventor(s): Otto Yang, Ayub Ali, Don Diamond

Keywords(s): Gene Editing Systems, Gene Therapy, Therapeutics & Vaccines

Category(s): Therapeutics > Gene Therapy And Editing

2019-958 SELECTIVE HDAC INHIBITORS

UCLA researchers in the Department of Chemistry and Biochemistry have synthesized a series of novel hydroxamic acids and derivatives as selective histone deacetylase (HDAC) inhibitors from simple starting materials.BACKGROUND:Histone deacetylase (HDAC) inhibitors are chemical compounds that regulates gene expression by inhibiting histone deacetylases....

Published: 7/19/2023 | Inventor(s): Michael Jung, Jonghoon Kim

Keywords(s): Chemical Manufacturing & Industrial/Bulk Chemicals, Cns, Gene Editing Systems, Gene Therapy, Infectious Diseases, Inflammatory Diseases, Therapeutics & Vaccines

Category(s): Therapeutics > CNS and Neurology, Therapeutics > Infectious Diseases, Therapeutics > Gene Therapy And Editing, Therapeutics > Inflammation And Inflammatory Diseases, Chemical > Industrial & Bulk Chemicals

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