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Longer-lived Mouse Models for Studying Gaucher Disease

The invention is a novel longer-lived mouse model for Gaucher disease. Gaucher disease is a genetic disorder that results from deficiencies in the enzyme glucocerebrosidase (GBA). The use of animal models to study how the disease progresses has been invaluable in research of this disorder. However, existing mouse models have been limited due to early...

Published: 7/25/2024 | Inventor(s): Mary Lamarca, Edward Ginns

Keywords(s): ANIMAL, DISORDERS, Gaucher, Gaucher Disease, Human, IDXXXX, Inherited, Models, RXXXXX, TRANSGENIC, VHXXXX, VPXXXX, WIXXXX, WJXXXX, WKXXXX, WMXXXX, XEXXXX

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

Stable, High-Yield Production of DT390-EGF Fusion Protein for Treatment of EGF-Receptor-Positive Cancers

This invention relates to the stable and high-yield production of a high-potency toxin protein called DT390-EGF. This toxin was developed for the treatment of EGF-receptor-positive cancers, including bladder cancer. Initial methods for synthesizing DT390-EGF relied on the use of E. coli. However, the production in E. coli was difficult to prepare and...

Published: 7/25/2024 | Inventor(s): Jung-hee Woo, David Neville, Arthur Frankel, Andrew Thorburn, Michael Glode, Thomas Flaig

Keywords(s): cancers, DT390-EGF, EGF, Fusion, POSITIVE, Protein, RECEPTOR, treatment, VCXXXX, WKXXXX, XEXXXX

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

Stopping Neurogenesis in Transgenic Mice and Rat Models that Express the HSV-thymidine kinase Gene in Neuronal Precursor Cells

This invention relates to novel mouse and rat models that permit the temporal death of neuronal precursor cells at any time point. Other existing methods of decreasing neurogenesis are relatively non-specific (e.g., injecting glucocorticoids) or require expensive equipment (e.g., focal x-irradiation) These mice and rats are being used to inhibit adult...

Published: 7/25/2024 | Inventor(s): James Pickel, Heather Cameron

Keywords(s): Driving, Expression, GFAP, HSV, Kinase, Listed LPM Maddox as of 4/15/2015, Mice, Post LPM Assignment Set 20150420, Pre LPM working set 20150418, PROMOTER, RATS, Thymidine, TRANSGENIC, VEXXXX, WIXXXX, XEXXXX

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

Generation of mutant mouse alleles that functionally disrupt production of BDNF from its ndividual promoters

This technology relates to a mouse model that improves an existing method of disrupting the production of the BDNF protein in specific parts of the brain. A current avenue of research seeks to examine how gene expression may effect long-lasting changes in the nervous system. Previous work has resulted in a mouse line in which the production of BDNF...

Published: 7/25/2024 | Inventor(s): Keri Martinowich

Keywords(s): ALLELES, AXXXXX, BDNF, DISRUPT, FUNCTIONALLY, Generation, Indiividual, Mouse, MUTANT, NC4XXX, NCXXXX, production, PROMOTERS, That, VEXXXX, WIXXXX, XEXXXX

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

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

Prematurely-Graying Mouse Line Demonstrates Regulation of Melanocyte Stem Cell Development by SOX10 (Sry-Related HMG-Box) Transcription Factor for Use in Regenerative Medicine

This technology includes transgenic mice to be used in the study of melanocyte stem cells (MSCs) for utilization in regenerative medicine. Using the melanocyte system as a model, we investigated establishment of MSCs in the hair bulge - the stem cell compartment of the hair. During embryogenesis, all melanoblasts express SOX10, but this expression is...

Published: 7/25/2024 | Inventor(s): Melissa Harris, William ("Bill") Pavan

Keywords(s): Cell, Demonstrates, Development, factor, HMG-Box, Line, Listed LPM Maddox as of 4/15/2015, Melanocyte, Mouse, Post LPM Assignment Set 20150420, Pre LPM working set 20150418, Prematurely-Graying, REGULATION, RXXXXX, SOX10, Sry-Related, Stem, transcription, VPXXXX, WIXXXX, XEXXXX

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

Murine Model of Niemann-Pick Disease Type C

This technology includes a transgenic mouse model of Niemann-Pick Disease Type C (NPC), which is a rare neurodegenerative disorder, characterized by intracellular accumulation of cholesterol and gangliosides. The mouse strain, Tg(Npcl), expresses wild-type NPC1 gene under the control of the prion promoter. When combined with the NPC deficient mouse...

Published: 7/25/2024 | Inventor(s): Stacie Loftus, William ("Bill") Pavan

Keywords(s): C, Disease, Listed LPM Maddox as of 4/15/2015, Model, Murine, NIEMANN-PICK, Post LPM Assignment Set 20150420, Pre LPM working set 20150418, RXXXXX, VEXXXX, VHXXXX, VNXXXX, VPXXXX, WIXXXX, WKXXXX, XEXXXX

Category(s): TherapeuticArea > Infectious Disease, TherapeuticArea > Psychiatry/Mental Health, Application > Therapeutics, TherapeuticArea > Endocrinology, TherapeuticArea > Dental, TherapeuticArea > Oncology, TherapeuticArea > Cardiology, TherapeuticArea > Neurology, Application > Research Materials, TherapeuticArea > Ophthalmology

Mouse Model of Hutchinson-Gilford Progeria Syndrome (HGPS) and Vascular Abnormalities (G608G) mutated form of human LNMA) for Therapeutic Development

Children with Hutchinson-Gilford progeria syndrome (HGPS) suffer from acceleration of certain aging symptoms, mainly cardiovascular disease that generally leads to death from myocardial infarction and/or stroke. The cause of HGPS has been discovered to be a de novo point mutation in lamin A (LNMA) gene. NHGRI Scientist have generated a transgenic...

Published: 7/25/2024 | Inventor(s): Francis Collins, Maria Eriksson, Renee Varga, Michael Erdos

Keywords(s): Abnormalities, HGPS, Hutchinson-Gilford, Listed LPM Maddox as of 4/15/2015, Model, Mouse, Post LPM Assignment Set 20150420, Pre LPM working set 20150418, Progeria, RXXXXX, Syndrome, vascular, VEXXXX, VPXXXX, XEXXXX

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

Lymphoblastoid Cell Lines with a Specific Allele of ABCA7 Gene for the Screening of Small Molecules for Therapeutic Development

This technology includes lymphoblastoid cell lines from individuals genotyped as carrying the minor (G) allele of ABCA7 SNP rs113809142 [ss491752998; SNV-chr19-1007244], to be used for small molecule screening and eventual therapeutic development. The ABCA7 gene is the ATP-binding cassette, sub-family A (ABC1), member 7. It encodes a protein that is...

Published: 7/25/2024 | Inventor(s): James ("Jim") Mullikin, Jennifer Johnston, Leslie Biesecker

Keywords(s): ABCA7, ALLELE, Cell, Gene, Lines, Listed LPM Contreras as of 4/15/2015, Lymphoblastoid, Post LPM Assignment Set 20150420, Pre LPM working set 20150418, RXXXXX, Specific, VHXXXX, VPXXXX, WIXXXX, WJXXXX, XEXXXX

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

DNA Methylation Based Non-invasive Blood Diagnostic Assay for Precision Cancer Detection and Classification

This technology includes a panel of 46 genomic loci of DNA methylation (represented by CpG dinucleotides on different chromosomes) with application in blood-based cancer screening. The markers robustly distinguish tumor from normal samples using 8 loci and classify 13 different tumor types. Using 39 loci, inventors were able to discriminate between...

Published: 7/25/2024 | Inventor(s): Gennady Margolin, Laura Elnitski

Keywords(s): assay, Based, Blood, CANCER, CLASSIFICATION, Detection, diagnostic, DNA, Humans, Methylation, NON-INVASIVE, PRECISION, VCXXXX, WBXXXX, WIXXXX, XEXXXX

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

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