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Minibody for Conditioning prior to Hematopoietic Stem Cell and Progenitor Cell Transplantation

Patient conditioning is a critical initial step in hematopoietic stem and progenitor cell (HSPC) transplantation procedures to enable marrow engraftment of infused cells. Conditioning regimens have traditionally been achieved by delivering cytotoxic doses of chemotherapeutic agents and radiation. However, these regimens are associated with significant...

Published: 7/25/2024 | Inventor(s): Daisuke Araki, Diogo Magnani, Zhirui Wang, Andre Larochelle

Keywords(s): 1CXXXX, 2IXXXX, 2JXXXX, Anti-cMPL, Cells, DEPLETION, Divalent, DT-390-Conjugated, hematopoietic, Progenitor, ScFv, Stem, VCXXXX, VPXXXX, WJXXXX, WMXXXX, XAXXXX

Category(s): TherapeuticArea > Ophthalmology, TherapeuticArea > Endocrinology, TherapeuticArea > Dental, TherapeuticArea > Oncology, TherapeuticArea > Cardiology, Application > Consumer Products, Application > Therapeutics, TherapeuticArea > Infectious Disease, TherapeuticArea > Immunology, ResearchProducts > Antibodies

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

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

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

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

Gene Therapy for Cobalamin C Deficiency (cblC) with Viable Mouse Models

Cobalamin C deficiency (cblC) is the most common inborn error of intracellular cobalamin metabolism and is caused by mutations in MMACHC, a gene responsible for processing and trafficking dependent enzymes: intracellular cobalamin, resulting in elevated methylmalonic acid and homocysteine and methionine deficiency. Disease manifestations include growth...

Published: 7/25/2024 | Inventor(s): Jennifer Sloan, Charles Venditti

Keywords(s): C, cblC, Cobalamin, DEFICIENCY, Gene, THERAPY, VDXXXX, VEXXXX, VPXXXX, WIXXXX, WJXXXX, XEXXXX

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

Novel Codon-Optimized MUT Gene Therapeutic for Methylmalonic Acidemia (MMA)

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): Colin Shea, Ciprian Crainiceanu, Elizabeth Sweeney, Russell Shinohara, Arthur Goldsmith, Daniel Reich

Keywords(s): Automatic, brain, Detection, IMAGING, Incidence, Lesion, Listed LPM Shmilovich as of 4/15/2015, Longitudinal, Magnetic, MULTIMODALITY, Post LPM Assignment Set 20150420, Pre LPM working set 20150418, Resonance, software, SuBLIME:, VPXXXX, WJXXXX, XEXXXX

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

Reducing Bloodstream Neutrophils as a Treatment for Lung Infection and Inflammation

During lung infection, bloodstream neutrophils (PMNs) responding to infection travel to the airspace lumen. Although successful arrival of microbicidal PMNs to the airspace is essential for host defense against inhaled pathogens, excessive accumulation of PMNs in the lung contributes to the pathogenesis of several prevalent lung disorders, including...

Published: 7/25/2024 | Inventor(s): Carmen Williams, Wan-Chi Lin, Michael Fessler

Keywords(s): 2, BARRIER, DA1XXX, DA3XXX, DA4XXX, DAXXXX, DBXXXX, During, DXXXXX, Epithelial, Infection, Inflammation, INTEGRITY, LUNG, MEMBRANE, PROTECT, Protein, respiratory, Targeting, VJXXXX, VKXXXX, VLXXXX, VPXXXX, WBXXXX, WJXXXX, WKXXXX, XAXXXX, YAXXXX, YBXXXX, YCXXXX

Category(s): Collaboration Sought > Collaboration, ResearchProducts > Antibodies, Application > Diagnostics, Application > Therapeutics, TherapeuticArea > Ophthalmology, TherapeuticArea > Endocrinology, TherapeuticArea > Infectious Disease, TherapeuticArea > Cardiology, TherapeuticArea > Dental, TherapeuticArea > Immunology, TherapeuticArea > Oncology

Heartland Virus Humanized Monoclonal Antibodies for Diagnostic and Therapeutic Development

Heartland virus (HRTV) is a novel tick-borne virus first discovered in 2009 that causes flu-like symptoms such as fever, headaches, fatigue, muscle aches, and diarrhea. Patients with HRTV often have low white blood cell counts, low platelet counts, and abnormal liver function tests which can become severe. Cases of Heartland virus disease have been...

Published: 7/25/2024 | Inventor(s): Aaron Brault, Amanda Calvert

Keywords(s): antibodies, Diagnostics, heartland, Listed LPM Houze as of 4/15/2015, monoclonal, NCEZID, NCEZID-DVBD, Post LPM Assignment Set 20150420, Pre LPM working set 20150418, Specific, therapeutics, virus, WBXXXX, WFXXXX, WJXXXX, XAXXXX, YAXXXX, YBXXXX, YCXXXX, YFXXXX

Category(s): Collaboration Sought > Collaboration, Collaboration Sought > Licensing, TherapeuticArea > Immunology, ResearchProducts > Antibodies, Application > Occupational Safety and Health, Application > Therapeutics, Application > Consumer Products, Application > Diagnostics

Regenerative Therapy for Cartilage Damage

The subject technology discloses a tissue engineering method for treating cartilage damage. Stem cells from bone marrow are attached to a novel scaffold, hyaluronic acid-coated fibrin microbeads, and transferred to a live host to form cartilage. The regenerated cartilage is stable and not hypertrophic, which has been problematic in the current regenerative...

Published: 8/21/2024 | Inventor(s): Mitchell Shirvan, Rafael Gorodetsky, Sergei Kuznetsov, Astar Lazmi-Hailu, Pamela Robey

Keywords(s): Bone, Cartilage, Cells, Fibrin, Generation, Human, Hyaline, Marrow, Microbeads, STABLE, Stromal, TRANSPLANTATION, VAXXXX, Vivo, WJXXXX, XCXXXX, XDXXXX, YAXXXX, YBXXXX, YCXXXX

Category(s): Application > Diagnostics, Application > Research Materials, Application > Non-Medical Devices, Application > Therapeutics, TherapeuticArea > Immunology, TherapeuticArea > Geriatrics, Application > Medical Devices

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