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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

A Genetic System in Yeast for Functional Identification of Human p53 Mutations

Mutations in the p53 gene are associated with 50% of all cancers and nearly 80% of the p53 mutations are missense changes. We have developed genetic assays based in yeast that can functionally categorize expressed p53 mutant proteins. The combined assays are referred to as the FIP53 system. Because human p53 cDNA can be conveniently cloned in yeast,...

Published: 7/25/2024 | Inventor(s): Alberto Inga, Michael Resnick

Keywords(s): CA1XXX, CAXXXX, FUNCTIONAL, GAXXXX, Genetic, Human, Identification, Listed LPM Wong as of 4/15/2015, Mutations, p53, Patent Category - Biotechnology, Post LPM Assignment Set 20150420, Pre LPM working set 20150418, SACGHS DNA Patent Initial Set, System, X, Yeast

Category(s): Collaboration Sought > Licensing, TherapeuticArea > Oncology, Application > Diagnostics, 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

Rapid and Sensitive Detection of Nucleic Acid Sequence Variations

The ability to easily detect small mutations in nucleic acids, such as single base substitutions, can provide a powerful tool for use in cancer detection, perinatal screens for inherited diseases, and analysis of genetic polymorphisms such as genetic mapping or for identification purposes. Current approaches make use of the mismatch that occurs between...

Published: 4/8/2024 | Inventor(s): Kenji Adzuma, Kiyoshi Mizuuchi, Katsuhiko Yanagihara

Keywords(s): (4)r syndrome, AC6XXX, ACXXXX, AXXXXX, C syndrome, CA1AXX, CA1XXX, CAXXXX, Chromosome 4 ring syndrome, Chromosome 6 ring syndrome, Chromosome 7 ring syndrome, CXXXXX, Cystic fibrosis, Detection, DNA, Fragile X syndrome, G syndrome, Genetic, Huntington disease, Huntington disease; Huntington's disease, Hypertelorism with esophageal abnormality and hypospadias, IA6XXX, IAXXXX, Insertion, IXXXXX, Method, Mismatch-Targeted, Muscular dystrophy, Mutations, N syndrome, R(6) syndrome, R(7) syndrome, Syndrome X, Transposon, W syndrome, W syndrome; Syndrome W

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

A Full-Length Infectious cDNA Clone of Zika Virus from the 2015 Epidemic in Brazil as a Genetic Platform for Studies of Virus-Host Interactions and Vaccine Development

An arthropod-borne virus, Zika virus (ZIKV), has recently emerged as a major human pathogen. Associated with complications during perinatal development and Guillain-Barré syndrome in adults, ZIKV raises new challenges for understanding the molecular determinants of flavivirus pathogenesis. This underscores the necessity for the development of a reverse...

Published: 7/25/2024 | Inventor(s): Konstantin Tsetsarkin, Alexander Pletnev

Keywords(s): 2015, Brazil, cDNA, CLONE, DAXXXX, DBXXXX, DC5BXX, DC5XXX, DC6XXX, DCXXXX, DD1XXX, DDXXXX, Development, DXXXXX, Epidemic, FULL-LENGTH, Genetic, INFECTIOUS, INTERACTIONS, PLATFORM, STUDIES, Vaccine, virus, Virus-Host, Zika

Category(s): Collaboration Sought > Collaboration, Application > Therapeutics, TherapeuticArea > Infectious Disease, Application > Vaccines, Application > Research Materials, Application > Diagnostics