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Cloning and Characterization of an Avian Adeno-Associated Virus and Uses Thereof

Currently, adeno-associated virus (AAV) represents the gene therapy vehicle of choice because it has many advantages over current strategies for therapeutic gene insertion. AAV is less pathogenic than other virus types; stably integrates into dividing and non-dividing cells; integrates at a consistent site in the host genome; and shows good specificity...

Published: 7/25/2024 | Inventor(s): John (Jay) Chiorini, Ioannis Bossis

Keywords(s): GB2A2X, GB2AXX, GB2XXX, GB3XXX, GBXXXX, GXXXXX

Category(s): Collaboration Sought > Collaboration, Collaboration Sought > Licensing, Application > Therapeutics

Novel Codon-Optimized Gene Therapeutic for Methylmalonic Acidemia

Methylmalonic Acidemia (MMA) is a metabolic disorder characterized by increased acidity in the blood and tissues due to toxic accumulation of protein and fat by-products resulting in seizures, strokes, and chronic kidney failure. A significant portion of MMA cases stem from a deficiency in a key mitochondrial enzyme, methylmalonyl-CoA mutase (MUT),...

Published: 7/25/2024 | Inventor(s): Randy Chandler, Charles Venditti

Keywords(s): Acidemia, Class, GB2AXX, GBXXXX, GXXXXX, Methylmalonic, Methylmalonyl-CoA, MMA, MUT, Mutase, Synthetic, TRANSGENE, treatment

Category(s): Collaboration Sought > Collaboration, Application > Therapeutics

Diagnostics, Vaccines, and Delivery-Vehicles Related to Novel Phlebovirus

This CDC invention relates to primers and probes that specifically hybridize with Heartland virus (HRTLDV), a unique member of the genus Phlebovirus. It further relates to polyclonal antibodies specific for HRTLDV proteins. Serological detection assays using HRTLDV nucleic acid molecules, proteins, probes, primers, and antibodies are provided. Importantly,...

Published: 7/25/2024 | Inventor(s): Stuart Nichol, William Nicholson, Cynthia Goldsmith, Aubree Kelly, Laura McMullan

Keywords(s): AC4XXX, ACXXXX, ANTIBODY, ANTIGEN, AXXXXX, CDC Docket Import, CDC Docket Import CDC Prosecuting, Compositions, DA4BXX, DA4XXX, DAXXXX, DC1XXX, DC5BXX, DC5XXX, DCXXXX, DDXXXX, Delivery, delivery system, DEVELOPED, diagnostic, DXXXXX, GB2CXX, GB2XXX, GBXXXX, GCXXXX, GENE THERAPY, gene transfer, GXXXXX, heartland, heartland virus, Hospitalized, Identification, ISOLATES, Isolation, Methods, Missouri, Novel, PATHOGENIC, PHLEBOVIRUS, PLATFORM, Platforms, sequence, therapeutic, therapeutic candidates, THEREOF, TICK, TICK-BORNE, TICKS, TWO, Vaccine, VACCINE VECTORS, Vector, Vector-based, Vector-borne, Vector-borne diseases, VETERINARY, VETERINARY vaccine, viral, virus, VLXXXX, VOXXXX, WBXXXX, WDXXXX, WFXXXX, WIXXXX, WJXXXX, XAXXXX, XCXXXX, XHXXXX, YAXXXX, YBXXXX, Zoo

Category(s): Collaboration Sought > Licensing, ResearchProducts > Antibodies, ResearchProducts > Research Equipment, Application > Research Materials, TherapeuticArea > Immunology, Application > Diagnostics, Application > Vaccines, Application > Therapeutics, TherapeuticArea > Infectious Disease, Application > Occupational Safety and Health, Application > Consumer Products

Small Interfering RNA Inhibition of Cannabanoid-1 Receptor (CB1R) for Treating Type 2 Diabetes

The invention pertains to the use of glucan encapsulated non-immunostimulatory small interfering RNAs (siRNAs) to treat type-2 diabetes. Endocannabinoids (EC) are lipid signaling molecules that act on the same cannabinoid receptors that recognize and mediate the effects of endo- and phytocannabanoids. EC receptor CB1R activation is implicated in the...

Published: 7/25/2024 | Inventor(s): Tony Jourdan, Michael Czech, Myriam Aouadi, George Kunos

Keywords(s): CB1, DIABETES, GB1AXX, GB1XXX, GBXXXX, GXXXXX, IBXXXX, IXXXXX, Knockdown, MACROPHAGES, RECEPTORS, SELECTIVE, SiRNA-mediated, treatment

Category(s): Collaboration Sought > Licensing, Application > Research Materials, Application > Diagnostics, Application > Therapeutics

Adeno-Associated Virus Gene Therapy for Diabetes and Obesity

This invention is directed to adeno-associated virus (AAV) vector delivery of exendin-4 (Ex-4) to salivary glands as treatment for diabetes and obesity. Ex-4 is a potent and long-acting agonist of the receptor for glucagon-like peptide 1 (GLP-1). Scientists at NIDCR have shown that AAV-mediated delivery of Ex-4 resulted in improved glucose homeostasis...

Published: 7/25/2024 | Inventor(s): Giovanni Di Pasquale, Edward Mannucci, John (Jay) Chiorini

Keywords(s): DIABETES, EPITHELIA, Expression, Fusion, GBXXXX, Gland, IBXXXX, Listed LPM Contreras as of 4/15/2015, NGF/exendin-4, Novel, Post LPM Assignment Set 20150420, Pre LPM working set 20150418, Protein, Salivary, treatment

Category(s): Application > Therapeutics

Modulating Autophagy as a Treatment for Lysosomal Storage Diseases

Researchers at NIAMS have developed a technology for treatment of lysosomal storage diseases by inhibition of autophagy. Pompe disease is an example of a genetic lysosomal storage disease caused by a reduction or absence of acid alpha-glucosidase (GAA). Patients with Pompe disease have a lysosomal buildup of glycogen in cardiac and skeletal muscle...

Published: 8/14/2024 | Inventor(s): Cynthia Schreiner, Paul (Estate of) Plotz, Shoichi Takikita, Tao Xie, Rebecca Baum, Nina Raben

Keywords(s): Acid, A-glucosidase, Autophagy, B, Clearance, Complete, DEFICIENCY, Disabling, Disease, Enzyme, ERT, GB1XXX, GBXXXX, Genetic, GLYCOGEN, GXXXXX, IBXXXX, Inactivation, IXXXXX, Lysosomal, Model, Mouse, MUSCLE, Near, Patent Category - Biotechnology, Permits, Pompe, REPLACEMENT, SKELETAL, Storage, STORED, THERAPY, UA1XXX

Category(s): Collaboration Sought > Collaboration, Application > Therapeutics, TherapeuticArea > Rare / Neglected Diseases, Application > Diagnostics, Application > Research Materials

Modulation of Leucine-rich Repeats and Calponin Homology Domain-containing Protein 4 (Lrch4) Activity for Therapeutic Applications

NIH Inventors have recently discovered a novel Leucine-rich repeat and calponin homology domain-containing protein 4 (Lrch4) in a proteomic screen of the plasma membrane of lipopolysaccharide (LPS)-exposed macrophages. Expression data by RT-PCR revealed that all Lrch family members (1-4) are expressed in macrophages, but only Lrch4 was recruited into...

Published: 7/25/2024 | Inventor(s): Michael Fessler

Keywords(s): CB6XXX, CBXXXX, CXXXXX, DISEASES, GB1XXX, GBXXXX, GXXXXX, IB3XXX, IBXXXX, INFLAMMATORY, IXXXXX, Lrch4, Sepsis, TARGET, therapeutic

Category(s): Collaboration Sought > Collaboration, Application > Research Materials, Application > Diagnostics, Application > Therapeutics, TherapeuticArea > Oncology, TherapeuticArea > Immunology

Identification of EGFR as A Receptor for AAV6 Transduction

AAV vectors offer unique advantages in gene therapy applications. Studies have shown that these replication deficient parvovirus vectors can deliver DNA to specific tissues and confer long-term transgene expression in a variety of systems. Although many studies have looked at the tissue-specific expression elicited by each of the AAV serotypes, a...

Published: 7/25/2024 | Inventor(s): Michael Schmidt, Melodie Weller, John (Jay) Chiorini

Keywords(s): AAV6, EGFR, GB2A2X, GB2AXX, GB2XXX, GBXXXX, GXXXXX, Identification, RECEPTOR, transduction

Category(s): Application > Therapeutics

Methods to Increase Stability of Recombinant Vaccinia-Vectored Vaccines and Increase Expression of a Foreign Gene Inserted in Such Vaccines

The technology offered for licensing is in the field of vaccinia-based recombinant vaccines. In particular the invention relates to methods of stabilizing the recombinant virus, thus resulting in efficient production of the vaccine and efficient expression of the inserted gene. Stabilization of the recombinant virus is achieved by the insertion of...

Published: 7/25/2024 | Inventor(s): Patricia Earl, Linda Wyatt, Bernard Moss

Keywords(s): ANKARA, Del, Expression, Fied, Foreign, GB1DXX, GB1XXX, GBXXXX, Gene, GENES, GXXXXX, INCREASE, Insertion, LIl, Modi, Modified, MVA, Patent Category - Biotechnology, PLASMID, SHUTTLE, SITE, STABILITY, vaccinia, Vector, virus

Category(s): Collaboration Sought > Licensing, Application > Therapeutics

Scalable Purification of AAV2, AAV4 or AAV5 Using Ion-Exchange Chromatography

Adeno-associated viruses (AAVs) constitute, as a group, the vehicle of choice for gene therapy because of several attractive features. Among others, AAVs are less pathogenic than other viruses, and they can be used for the long-term expression of therapeutic genes. This invention describes a simple ion-exchange (HPLC) methodology to purify different...

Published: 7/25/2024 | Inventor(s): John (Jay) Chiorini, Nikola Kaludov

Keywords(s): AAV2, AAV4, AAV5, CHROMATOGRAPHY., GB2DXX, GB2XXX, GBXXXX, GXXXXX, ION-EXCHANGE, purification, SCALABLE

Category(s): Application > Therapeutics

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