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Search Results - gene+therapy
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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
AAV-Based Treatment of Guanidinoacetate Methyltransferase (GAMT) Deficiency (UCLA Case No. 2021-012)
SUMMARY: UCLA researchers in the Departments Molecular & Medical Pharmacology and Surgery in the David Geffen School of Medicine have developed a novel and tissue-specific
gene therapy
treatment for Guanidinoacetate methyltransferase (GAMT) deficiency. BACKGROUND: Guanidinoacetate Methyltransferase (GAMT) deficiency is caused by a mutation in...
Published: 8/10/2023
|
Inventor(s):
Gerald Lipshutz
Keywords(s):
Adeno-associated viruses (AAV)
,
Creatine
,
GAMT deficiency
,
Gene Therapy
,
Metabolic Diseases
Category(s):
Therapeutics >
Gene Therapy
And Editing
,
Therapeutics > Genetic Diseases
,
Therapeutics > Metabolism And Endocrinology
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
2021-030 BIODEGRADABLE MICRONEEDLE PATCH FOR TRANSDERMAL GENE DELIVERY
UCLA researchers in the Department of Bioengineering have developed a novel microneedle patch that effectively delivers nucleic acids across the skin for use as a delivery platform for gene therapies.BACKGROUNDGene therapies work by delivering designed corrective nucleic acid sequences to cells expressing mutant disease-causing forms of genes. For gene...
Published: 9/5/2023
|
Inventor(s):
Ali Khademhosseini
,
Wujin Sun
Keywords(s):
Gene Therapy
Category(s):
Therapeutics >
Gene Therapy
And Editing
2020-781
GENE THERAPY
VECTORS FOR TREATING LIMB GIRDLE MUSCULAR DYSTROPHY
Researchers in the Departments of Neurology at UCLA and the University of Washington have developed a novel AAV
gene therapy
that treats limb-girdle muscular dystrophy type R1/2A (LGMD2A), an as of yet unmet target.BACKGROUND:Muscular dystrophies are a group of muscle diseases caused by mutations in a persons’ genes. Mutations within the calpain3...
Published: 7/19/2023
|
Inventor(s):
Melissa Spencer
,
Irina Kramerova
,
Steve Hauschka
,
Jeffrey Chamberlain
Keywords(s):
Gene Therapy
,
Muscular Dystrophy
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: 7/19/2023
|
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: 7/19/2023
|
Inventor(s):
Otto Yang
,
Ayub Ali
,
Don Diamond
Keywords(s):
Gene Editing Systems
,
Gene Therapy
,
Therapeutics & Vaccines
Category(s):
Therapeutics >
Gene Therapy
And Editing
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