Search Results - gene+therapy

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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
Direct and Effective Method To Reverse The Effects of Fragile X Syndrome
A method of treating Fragile X syndrome by correcting the abnormal DNA clusters causing the characteristic symptoms. Problem: Fragile X syndrome (FXS) affects approximately 1 in 4,000 males and 1 in 8,000 females and is characterized by symptoms such as: Inherited Intellectual Disability Anxiety Attention Deficit Disorder (ADD) Skin, Ovaries...
Published: 3/8/2024   |   Inventor(s): Jennifer Phillips-Cremins, Linda Zhou, Chunmin GE
Keywords(s): Bioengineering, Gene Therapy, Genetic Disorders, Neurodegenerative Diseases, Neurology
Category(s): Technology Classifications > Therapeutics, Technology Classifications > Vaccines
HIGH-CAPACITY LENTIVIRAL VECTORS
HIGH-CAPACITY LENTIVIRAL VECTORS Researchers at Stanford have developed new lentiviral vectors that are able to deliver more genetic information than currently available vectors, aiding in the development of gene therapy. Gene therapy is one of the most promising therapeutic avenues in translational medicine. This method involves the genetic engineering...
Published: 8/17/2023   |   Inventor(s): Michael Fischbach, Brian Caliando, Gabriel Filsinger
Keywords(s): Gene Therapy, Lentiviral Vector
Category(s): Technology Classifications > Biology
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
Gene Therapy for Treatment of CRX-Autosomal Dominant Retinopathies
Abstract: Mutations in the cone rod homeobox (CRX) transcription factor lead to distinct retinopathy phenotypes, including early-onset vision impairment in dominant Leber congenital amaurosis (LCA). Adeno-Associated virus (AAV) vector-mediated delivery of a CRX cDNA under the control of a CRX promoter region partially restored photoreceptor phenotype...
Published: 4/8/2024   |   Inventor(s): Anand Swaroop, Kamil Kruczek, Suja Hiriyanna, Zhijian Wu
Keywords(s): AAV, Adeno-associated Virus, Cone-rod Dystrophy, CRD, GENE THERAPY, LCA, Leber Congenital Amaurosis, Lentivirus, NEI, rare disease, Retinitis Pigmentosa, Retinopathies, RP, Swaroop
Category(s): TherapeuticArea > Opthamology, TherapeuticArea > Eye / Ear / Nose / Throat, Collaboration Sought > Licensing, Application > Therapeutics, Collaboration Sought > Collaboration
Induced Pluripotent Stem Cells Derived from Patients with CEP290-associated Ciliopathies and Unaffected Family Members
Abstract: Approximately one-third of non-syndromic retinal dystrophies involve a defect in a ciliary protein. Non-syndromic retinal ciliopathies include retinitis pigmentosa, cone dystrophy, cone-rod dystrophy, macular dystrophy, and Leber-congenital amaurosis (LCA). Many CEP290-LCA patients also exhibit auditory and olfactory defects. Induced pluripotent...
Published: 4/8/2024   |   Inventor(s): Anand Swaroop, Hiroko Shimada-Ishii, Yu Holly Chen, Milton English
Keywords(s): Anosmia, Cell therapy, Ciliopathies, Congenital Blindness, Drug Development, GENE THERAPY, hearing loss, iPS, National Eye Institute, NEI, Pluripotent Stem Cells, Primary Cilia, Retina, Retinal degeneration, Swaroop, VISION
Category(s): Application > Research Materials, Collaboration Sought > Licensing, Collaboration Sought > Collaboration, TherapeuticArea > Opthamology, TherapeuticArea > Eye / Ear / Nose / Throat
Mouse Embryo Culture Chamber and Imaging System and Methods of Use
Abstract: The culture of mouse embryos ex utero and continuous monitoring and imaging of embryos as they develop have applications in drug testing, genetic studies, and basic research on embryonic development. However, the embryo culture systems currently available for post-implantation embryos include rolling bottle culture systems, which do not permit...
Published: 4/8/2024   |   Inventor(s): Vijay Kalaskar
Keywords(s): Culture Chamber, Embryo Development, Embryonic, FETAL, GENE THERAPY, IMAGING, In Vitro Drug Toxicology, Kalaskar, National Eye Institute, NEI, Organ System Therapy
Category(s): Application > Non-Medical Devices, TherapeuticArea > Reproductive Health, Application > Medical Devices, Collaboration Sought > Collaboration, Collaboration Sought > Licensing, TherapeuticArea > Opthamology, TherapeuticArea > Eye / Ear / Nose / Throat
Recombinant Virus Vectors for the Treatment of Glycogen Storage Disease type Ib (GSD-Ib)
Abstract: Glycogen storage disease type Ib (GSD-Ib) is an autosomal recessive disorder caused by deficiencies in glucose-6-phosphate transporter (G6PT), a ubiquitously expressed endoplasmic reticulum (ER) protein that translocates G6P from the cytoplasm into the ER lumen.  Inside the ER, G6P is hydrolyzed to glucose and phosphate by either the liver/kidney/intestine-restricted...
Published: 4/8/2024   |   Inventor(s): Janice Chou
Keywords(s): autosomal recessive disorder, Chou, G6PT, GENE THERAPY, glucose homeostasis, Glycogen storage disease type Ib, GSD-Ib, hepatocellular adenoma, metabolic and immune disorders, myeloid dysfunction, NEUTROPENIA, NICHD
Category(s): Collaboration Sought > Collaboration, Application > Therapeutics, Collaboration Sought > Licensing, TherapeuticArea > Endocrinology
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