Search Results - gene+therapy

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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: 12/20/2024   |   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
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: 11/22/2024   |   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
The First Curative Treatment of Phenylketonuria Using Genetic Engineering Approaches
The base editing of phenylalanine hydroxylase enzyme in the liver to restore phenylalanine metabolism and prevent neurotoxic effects. Problem: Phenylketonuria (PKU) is a severe disorder affecting approximately 1 in 10,000 U.S. newborns. PKU is marked by a genetic mutation in the phenylalanine hydroxylase enzyme (PAH), resulting in phenylalanine (Phe)...
Published: 10/28/2024   |   Inventor(s): Kiran Musunuru, Xiao Wang, Dominique Brooks
Keywords(s): Gene Editing, Gene Therapy, Genetic Disorders, Neurodegenerative Diseases, Neurology
Category(s): Technology Classifications > Therapeutics
Lipid Nanoparticles That Specifically Target The Lungs
Lung-targeting lipid nanoparticles allowing organ specific mRNA delivery for gene therapy applications. Problem: Gene therapy via mRNA delivery allows the rapid generation of therapeutics and vaccines for a wide range of applications. However, mRNA is especially vulnerable to degradation in the body, necessitating packaging within lipid nanoparticles....
Published: 10/17/2024   |   Inventor(s): Michael Mitchell, Lulu Xue
Keywords(s): Bioengineering, COVID-19, Gene Therapy, Infectious Disease, Pulmonary
Category(s): Technology Classifications > Chemical Processes and Synthesis, Technology Classifications > Therapeutics, Technology Classifications > Vaccines
Strategies for Mitigating Acute Side Effects of Lung-Targeted Nanocarriers
Approaches and Design Techniques to Alleviate Coagulation-Related Acute Side Effects of Lung-Targeted Nanocarriers Problem: Lipid nanoparticles (LNPs) play a crucial role in drug delivery, enabling targeted drug administration to specific tissues. LNPs tailored for lung-specific delivery often incorporate cationic (positively charged) lipids. Unfortunately,...
Published: 6/26/2024   |   Inventor(s): Jacob Brenner, Vladimir Muzykantov, Jacob Myerson, Oscar Marcos Contreras, Marco Zamora, Serena Omo-Lamai
Keywords(s): Bioengineering, Drug Delivery, Gene Editing, Gene Therapy, Nanotechnology, Platform Technology, Pulmonary
Category(s): Technology Classifications > Therapeutics
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
Silencing of PRLΔE1 expression: a gene-agnostic treatment for retinal degenerations
Viral vector-mediated gene silencing of a novel short retinal isoform of Prolactin promotes photoreceptor survival. Problem: Age-related macular degeneration (AMD) and inherited retinal degeneration (IRDs) have extreme mutational heterogeneity, and many IRDs are orphan diseases. Developing individual gene therapies for each causative gene is impractical...
Published: 7/2/2024   |   Inventor(s): William Beltran, Raghavi Sudharsan
Keywords(s): Drug Target, Gene Therapy, Genetic Disorders, Neurodegenerative Diseases, Ophthalmology, Orphan Disease
Category(s): Technology Classifications > Therapeutics
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