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Viral Vector-Based
Gene Therapy
for OPA1 Gene Mutation-Induced Dominant Optic Atrophy (UCLA Case No. 2025-004)
UCLA researchers in the Department of Ophthalmology have developed a viral vector-based
gene therapy
to restore OPA1 protein levels in patients with OPA1-mutated dominant optic atrophy. The optimized vectors will be delivered intravitreally to patients’ eyes, providing a minimally invasive, effective treatment with reduced side effects. BACKGROUND:...
Published: 3/6/2025
|
Inventor(s):
Xianjie Yang
Keywords(s):
Adeno-associated viruses (AAV)
,
disease model
,
dominant optic atrophy
,
Gene Therapy
,
intravitreal delivery
,
Lentivirus Viral Vector
,
mitochondria
,
mutation
,
Neuron
,
OPA1
,
Ophthalmology
,
pluripotent stem cell (PSC)
,
retinal ganglion cell
Category(s):
Therapeutics > CNS and Neurology
,
Therapeutics >
Gene Therapy
And Editing
,
Therapeutics > Ophthalmology
Recombinant Adeno-Associated Virus 8 Human Solute Carrier Family 4 Member 11 (AAV8-hSLC4A11) for Congenital Hereditary Endothelial Dystrophy (CHED) (UCLA Case No. 2025-096)
UCLA researchers from the Department of Ophthalmology have developed a novel
gene therapy
method to treat Congenital Hereditary Endothelial Dystrophy. BACKGROUND: Congenital Hereditary Endothelial Dystrophy (CHED) is a rare autosomal recessive disease that affects corneal endothelial cells, which can lead to blindness if left untreated. CHED is more...
Published: 2/25/2025
|
Inventor(s):
Anthony Aldave
Keywords(s):
Gene Therapy
Category(s):
Therapeutics > Ophthalmology
,
Therapeutics >
Gene Therapy
And Editing
Gene Therapy
for DOCK8 Deficiency (UCLA Case No. 2023-178)
UCLA researchers in the Department of Pediatrics Allergy and Immunology have developed a
gene therapy
method to express the full-length DOCK8 protein in cells, aimed at treating DOCK8 deficiency. These method allows for safe and effective DOCK8 expression in hematopoietic stem cells without the need for an HLA-matched donor, enabling patients to undergo...
Published: 2/18/2025
|
Inventor(s):
Caroline Kuo
Keywords(s):
bone marrow
,
bone marrow transplants
,
DOCK8
,
DOCK8 deficiency
,
Gene Editing Systems
,
Gene Therapy
,
hematopoietic stem cells
,
Immune System
,
immunodeficiency
,
Immunology
,
intein splicing
Category(s):
Therapeutics > Immunology And Immunotherapy
,
Therapeutics >
Gene Therapy
And Editing
,
Therapeutics > Stem Cells And Regenerative Medicine
,
Life Science Research Tools > Plasmids/Vectors
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: 2/14/2025
|
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: 2/14/2025
|
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: 2/14/2025
|
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: 2/14/2025
|
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
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: 2/14/2025
|
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: 2/14/2025
|
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: 3/6/2025
|
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
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