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Retroviral Vector Packaging Cell Lines and Purification Methods for Gene Therapy

This invention relates to a novel gammaretroviral vector packaging cell line and a method of producing gammaretroviral vectors suitable for gene therapy. The described vectors may contain the gibbon ape leukemia virus (GALV) envelope with a CD11D8 epitope tag enabling their purification on a monoclonal antibody conjugated column. These vectors have...

Published: 7/25/2024 | Inventor(s): Wenqin Xu, Maribeth Eiden

Keywords(s): Cell, Gammaretroviral, GB2A2X, GB2CXX, Genetic, Line, Listed LPM Maddox as of 4/15/2015, Method, Novel, Packaging, Post LPM Assignment Set 20150420, Pre LPM working set 20150418, Producing, Suitable, THERAPY, Vector, vectors, VPXXXX, WIXXXX, WJXXXX, XEXXXX

Category(s): TherapeuticArea > Cardiology, TherapeuticArea > Ophthalmology, Application > Therapeutics, TherapeuticArea > Infectious Disease, TherapeuticArea > Dental, TherapeuticArea > Oncology, TherapeuticArea > Endocrinology, Application > Research Materials

Novel mouse models of methylmalonic acidemia (MMA) : C57Bl6/Sv129 Mut-/- (full knock-out) and (C57Bl6/Sv129) FvBN Mut -/- (full knock-out)

Methylmalonic acidemia (MMA) is an autosomal recessive disorder caused by the deficiency of the mitochondrial enzyme methylmalonyl-CoA mutase (MUT). It is characterized by metabolic instability, multiorgan pathology, and poor prognosis for long-term survival. To study MMA caused by MUT deficiency, a series of murine models have been constructed using...

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

Keywords(s): -/-, :, Acidemia, C57Bl6/Sv129, FULL, FvBN, KNOCK-OUT, Methylmalonic, MMA, Models, Mouse, MUT, Mut-/-, Novel, VPXXXX, WIXXXX, XEXXXX

Category(s): TherapeuticArea > Oncology, TherapeuticArea > Infectious Disease, TherapeuticArea > Ophthalmology, TherapeuticArea > Dental, Application > Research Materials, Application > Therapeutics, TherapeuticArea > Endocrinology, TherapeuticArea > Cardiology

Novel mouse model of mut- methylmalonic acidemia (MMA) Mut-/- Tg CBAMutG715V : Mut partial-deficiency

Methylmalonic acidemia (MMA) is an autosomal recessive disorder, caused by the deficiency of the mitochondrial enzyme methylmalonyl-CoA mutase (MUT). It is characterized by metabolic instability, multiorgan pathology, and poor prognosis for long-term survival. A well-characterized human mutation, p.G717V, has been introduced into mice. This mutation...

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

Keywords(s): :, Acidemia, CBAMutG715V, Methylmalonic, MMA, Model, Mouse, MUT, Mut-, Mut-/-, Novel, Partial-deficiency, Tg, VPXXXX, WIXXXX, XEXXXX

Category(s): TherapeuticArea > Ophthalmology, Application > Research Materials, TherapeuticArea > Cardiology, TherapeuticArea > Oncology, Application > Therapeutics, TherapeuticArea > Endocrinology, TherapeuticArea > Dental, TherapeuticArea > Infectious Disease

Novel mouse model of methylmalonic acidemia (MMA) Mut-/- Tg INS-Mck-Mut

Methylmalonic acidemia (MMA) is an autosomal recessive disorder, caused by the deficiency of the mitochondrial enzyme methylmalonyl-CoA mutase (MUT). It is characterized by metabolic instability, multiorgan pathology, and poor prognosis for long-term survival. Deletion of Mut in mice results in neonathal lethality, thus, to overcome this limitation,...

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

Keywords(s): Acidemia, INS-Mck-Mut, Methylmalonic, MMA, Model, Mouse, Mut-/-, Novel, Tg, VPXXXX, WIXXXX, XEXXXX

Category(s): TherapeuticArea > Cardiology, TherapeuticArea > Oncology, TherapeuticArea > Dental, TherapeuticArea > Endocrinology, Application > Therapeutics, TherapeuticArea > Ophthalmology, TherapeuticArea > Infectious Disease, Application > Research Materials

Novel Adeno-associated Viral (AAV) Vectors to Treat Hereditary Methylmalonic Acidemia (MMA) Caused by Methylmalonyl-coA Mutase (MMUT) Deficiency

Deficiency of the enzyme in methylmalonyl-CoA mutase (MMUT) results is a life-threatening disease, methylmalonic acidemia (MMA), that carries high rates of morbidity and mortality. NHGRI scientists have developed novel AAV vectors that combine the proprietary codon-optimized synMMUT alleles with either a liver-specific promoter from the human alpha-1...

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

Keywords(s): AAV, Acidemia, Adeno-associated, CAUSED, DEFICIENCY, Hereditary, Methylmalonic, Methylmalonyl-CoA, MMA, Mmut, Mutase, Novel, TREAT, vectors, viral, VPXXXX, WIXXXX, WJXXXX, XEXXXX

Category(s): TherapeuticArea > Cardiology, Application > Therapeutics, TherapeuticArea > Infectious Disease, TherapeuticArea > Ophthalmology, TherapeuticArea > Dental, TherapeuticArea > Oncology, TherapeuticArea > Endocrinology, Application > Research Materials

Aberrant Post-translational Modifications (PTMs) in Methyl- and Propionic Acidemia and the Construction of a Novel Sirtuin (SIRT) Gene to Metabolize PTMs

Isolated Methylmalonic Acidemia (MMA) and the related disorder Propionic Acidemia (PA) comprise a relatively common and heterogeneous group of inborn errors of metabolism. NHGRI scientist discovered that in isolated MMA, a novel inhibitory PTM, methylmalonyllysine, is generated and inactivates protein targets through the failure of SIRT-mediated deacylation,...

Published: 7/25/2024 | Inventor(s): PamelaSara Head, Charles Venditti

Keywords(s): Aberrant, Acidemia, Construction, Discovery, Ene, Metabolize, Methyl-, Modifications, Novel, Post-translational, Propionic, PTMs, Sirt, Sirtuin, VPXXXX, WIXXXX, WKXXXX, XEXXXX

Category(s): TherapeuticArea > Endocrinology, TherapeuticArea > Dental, TherapeuticArea > Oncology, TherapeuticArea > Cardiology, TherapeuticArea > Ophthalmology, Application > Therapeutics, TherapeuticArea > Infectious Disease, Application > Research Materials

Inhibition of Thioredoxin Reductase 1 (Trxr1) by Pyridine Compounds for Cancer Treatment

This technology includes the use of pyridines for anticancer treatment. A common feature of cancer cells is a high level of reactive oxygen species with a concomitant increase of two antioxidative systems to combat the toxicity: the glutathione and thioredoxin systems. Inhibiting either, or both, of these systems is a promising avenue to target cancer...

Published: 7/25/2024 | Inventor(s): Ajit Jadhav, Diane Luci, Thomas Dexheimer, Anton Simeonov, Nathan Coussens, David Maloney

Keywords(s): CANCER, Novel, PYRIDINES, Their, treatment, VCXXXX, WIXXXX, WKXXXX, XEXXXX

Category(s): Application > Research Materials, TherapeuticArea > Oncology, Application > Therapeutics

Novel ACRV1/ALK2 Inhibitors and Methods for Inhibiting BMP Signaling for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP)

This technology includes the identification and use of novel ACRV1/ALK2 inhibitors for the treatment of fibrodysplasia ossificans progressiva (FOP), an autosomal-dominant rare disease that affects one person in every 1-2 million. FOP is characterized by malformation of the great (big) toes during embryonic development and by progressive heterotopic...

Published: 7/25/2024 | Inventor(s): Jian-kang Jiang, Khalida Shamim, Philip Sanderson, Wei Zheng, Xiuli Huang, Gregory Tawa, Asaf Alimardanov, Arthur Lee, Junfeng Huang, Wenwei Huang

Keywords(s): ALK2, BMP, INHIBITING, Inhibitors, Methods, Novel, SIGNALING, VGXXXX, WIXXXX, WKXXXX, XEXXXX

Category(s): TherapeuticArea > Endocrinology, Application > Therapeutics, Application > Research Materials

Identification and Use of a Novel Functionally Selective GHSR1a Ghrelin Receptor Inhibitor, including NCGC00538279, for the Treatment of Food and Chemical Addiction

This technology includes a chemical series, including the NCGC00538279 compound, that selectively activates the GHSR1a G-protein pathway for calcium mobilization while only partially activating the beta-arrestin-2 translocation pathway. The resulting chemical series may be therapeutically valuable for addictive disorders. Activation of the GHSR1a G-protein...

Published: 7/25/2024 | Inventor(s): Noel Southall, Marc Ferrer-Alegre, Daniel Jansen, Mark Henderson, Xin Hu, David Kim, Juan Marugan

Keywords(s): ADDICTION, Chemical, FOOD, Funtionally, Ghrelin, LIGAND, NCG00538279, Novel, RECEPTOR, SELECTIVE, treatment, VEXXXX, VGXXXX, WIXXXX, WKXXXX, XEXXXX

Category(s): TherapeuticArea > Neurology, Application > Therapeutics, TherapeuticArea > Endocrinology, Application > Research Materials

Zika Virus NS1 Protein Monoclonal Antibodies for Research, Development, and Novel Diagnostics

Zika virus is a flavivirus that is spread by the bite of infected Aedes mosquitoes. The current outbreak and swift dissemination/spread of Zika virus (ZIKV) and its linkage to birth defects and neurological syndromes has prompted the development of novel diagnostic tests. Because ZIKV is serologically similar to other flaviviruses such as dengue virus...

Published: 7/25/2024 | Inventor(s): Dennis Bagarozzi, Jason Goldstein

Keywords(s): antibodies, Development, Diagnostics, monoclonal, NCEZID, NCEZID-DSR, Novel, virus, VLXXXX, WBXXXX, WIXXXX, XAXXXX, YBXXXX, Zika

Category(s): Collaboration Sought > Collaboration, Collaboration Sought > Licensing, Application > Diagnostics, Application > Research Materials, TherapeuticArea > Infectious Disease, TherapeuticArea > Immunology, ResearchProducts > Antibodies

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