Search Results - lysosomal

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Patient-derived induced pluripotent stem cell (iPSC) lines for the study of lysosomal storage diseases (LSDs)

This technology includes the generation and use of human induced pluripotent stem cell (iPSC) lines that can be used to study and screen potential therapeutics for lysosomal storage diseases (LSDs). LSDs are a group of 50 genetic disorders caused by mutations in the genes encoding lysosomal enzymes and proteins. Although various therapeutic approaches...

Published: 10/28/2024 | Inventor(s): Miao Xu, Yu-Shan Cheng, Manisha Pradhan, Jizhong Zou, Jeanette Beers, Wei Zheng

Keywords(s): Cell, COMPOUND, DERIVED, Disease, iPS, Lines, Lysosomal, Modeling, PATIENT, screening, Storage, VPXXXX, WIXXXX, XHXXXX

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

Novel Tocopherol and Tocopheryl Quinone Derivatives as Therapeutics for Lysosomal Storage Disorders

Novel tocopherol derivatives and tocopheryl quinone derivatives useful in the decrease of lysosomal substrate accumulation, the restoration of normal lysosomal size, and the treatment of lysosomal storage disorders (LSDs) are provided. The inventors have discovered that tocopherol and tocopheryl quinone derivatives with side chain modifications (such...

Published: 10/28/2024 | Inventor(s): Jingbo Xiao, Wei Zheng, John McKew, Juan Marugan

Keywords(s): ANALOGUES, Correctors, DISORDERS, IB6XXX, IBXXXX, IXXXXX, Lysosomal, Storage, Tocopherol

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

Cyclodextrins as Therapeutics for Lysosomal Storage Disorders

Cyclodextrins (CD), alone or in combination with other agents (e.g., vitamin E), as therapeutics for the treatment of lysosomal storage disorders (LSDs) caused by the accumulation of non-cholesterol lipids. CDs are sugar molecules in a ring form. The alpha-CD (6 sugars), beta-CD (7 sugars) and gamma-CD (8 sugars) are commonly used cyclodextrins. The...

Published: 10/28/2024 | Inventor(s): Wei Zheng, Miao Xu, Manju Swaroop, Juan Marugan, John McKew

Keywords(s): Combination, CYCLODEXTRIN, DISEASES, e, IB6XXX, IBXXXX, IXXXXX, Lysosomal, NB1JXX, NB1XXX, NBXXXX, NXXXXX, Storage, treatment, UA1XXX, USES, Vitamin

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

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