Search Results - gene+therapy

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Biocompatible And Low-Toxicity Lipid Nanoparticles (LNPs) For Selectively Delivering mRNA To The Bone Microenvironment
Ionizable LNPs with a rigid backbone of piperazine (PIP) linked to bisphosphonates (BPs) for the targeted delivery of mRNA to the bone. Problem: Available treatments for bone tissue related diseases provide only symptomatic relief and are associated with clinical challenges and potential side effects. Challenges persist in the delivery of therapeutic...
Published: 3/7/2025   |   Inventor(s): Michael Mitchell, Kyle Vining, Lulu Xue, Il-Chul Yoon
Keywords(s): Aging, Bioengineering, Drug Delivery, Gene Therapy, Orthopedics, Platform Technology, Women's Health
Category(s): Technology Classifications > Chemical Processes and Synthesis, Technology Classifications > Therapeutics
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
KIBRA-PKMzeta Interactions in LTP and Memory Maintenance
This technology offers the potential for developing novel and effective treatments for mood- and memory-related disorders and neuropathic pain. Background: During learning, neurons produce an enzyme called protein kinase M zeta (PKMzeta) which is responsible for (1) maintaining newly strengthened connections between neurons, and (2) memory. PKMzeta...
Published: 4/1/2025   |   Inventor(s): Todd Sacktor
Keywords(s): gene therapy, Technologies
Category(s): Campus > SUNY Downstate Health Sciences University, Technology Classifications > Drug Delivery, Technology Classifications > Healthcare, Technology Classifications > Therapeutics and Vaccines
A Method for Promoting Structural and Functional Rescue of Photoreceptors in the Eye to Treat Advanced Retinal Degeneration.
Discovery and use of retina-specific promoters to rescue photoreceptor function specifically at clinically relevant, advanced stages of disease. Problem: Despite the fact that retinal gene therapy studies have shown promising results in animal models, treatment of advanced retinal degeneration with currently available gene therapies have not been successful....
Published: 1/24/2025   |   Inventor(s): Raghavi Sudharsan, William Beltran
Keywords(s): Bioengineering, Gene Therapy, Genetic Disorders, Ophthalmology, Orphan Disease, Platform Technology, Regenerative Medicine
Category(s): Technology Classifications > Therapeutics
High-Throughput Approach to Identify New Lipid Nanoparticle Formulations for Vaccine and Drug Delivery
High-throughput in vivo screening method to identify lipid nanoparticle (LNP) formulations for local and systemic delivery for mRNA vaccines and cancer immunotherapies. Problem: Nucleic acid delivery is an attractive and rapidly growing area of research, particularly for the development of vaccines and cancer immunotherapy. Historically, viral vectors...
Published: 1/7/2025   |   Inventor(s): Michael Mitchell, Alex Hamilton
Keywords(s): Bioengineering, COVID-19, Drug Delivery, Gene Therapy, Immunology, Immunoncology, Immunotherapy, Infectious Disease, Nanotechnology
Category(s): Technology Classifications > Testing & Measurement, Technology Classifications > Vaccines, Technology Classifications > Therapeutics
Macrophage-Targeting Lipid Nanoparticles (MacLNPs) Deliver siRNA Intranasally to Treat Viral Pneumonia
siRNA-LNP knock-down of TAK1 delivered intranasally and to macrophages specifically prevents inflammation-induced pathology in a mouse model Problem: Macrophages are an important component of the innate immune system, but can mediate excessive inflammation and cause severe pathology if left unchecked. This poses a challenge for the treatment of acute...
Published: 12/19/2024   |   Inventor(s): Michael Mitchell, Lulu Xue, Andrew Vaughan, Gan Zhao
Keywords(s): Bioengineering, Drug Delivery, Gene Therapy, Immunology, Infectious Disease, Inflammation, Nanotechnology, Platform Technology
Category(s): Technology Classifications > Chemical Processes and Synthesis, Technology Classifications > Materials, Technology Classifications > Therapeutics, Technology Classifications > Vaccines
Targeted In Utero Therapeutics Delivery System for Precise Gene Editing
Targeted lipid nanoparticles (LNPs) that can deliver mRNA-based gene editing therapeutics to hematopoietic stem cells (HSCs) in utero Problem: Monogenic blood diseases, including sickle cell disease and / thalassemia, are among the most prevalent genetic conditions worldwide, causing significant pediatric morbidity with complications like painful...
Published: 1/2/2025   |   Inventor(s): Rohan Palanki, Michael Mitchell, William Peranteau
Keywords(s): Bioengineering, Gene Editing, Gene Therapy, Platform Technology
Category(s): Technology Classifications > Therapeutics
Space Shuttle-Inspired Branched Lipid Nanoparticles As Potent mRNA Transporters
Lipid nanoparticles (LNPs) with reversibly attached branched lipid tails for the delivery of mRNA for gene therapy. Problem: Delivery of mRNA to target cells enables the expression of engineered proteins but mRNA is unstable and negatively charged, necessitating a delivery vehicle to ensure its entry into cells. LNPs can ferry mRNA across the cell...
Published: 11/26/2024   |   Inventor(s): Michael Mitchell, Xuexiang Han
Keywords(s): Bioengineering, Drug Delivery, Gene Therapy, Nanotechnology, Obesity Metabolic Disease and Diabetes, Platform Technology
Category(s): Technology Classifications > Chemical Processes and Synthesis, Technology Classifications > Therapeutics
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