Search Results - niemann-pick

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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: 10/28/2024   |   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
Gene Therapy for Niemann-Pick Disease Type C
Investigators at the National Human Genome Research Institute (NHGRI) of the National Institutes of Health (NIH) are seeking collaborators to further develop gene therapy to treat Niemann-Pick Disease Type C (NPC). NPC is a rare, autosomal recessive, neurodegenerative disease. Approximately 95% of patients with NPC have mutations in NPC1, a gene implicated...
Published: 10/28/2024   |   Inventor(s): Randy Chandler, William ("Bill") Pavan, Charles Venditti
Keywords(s): Adeno-associated, C, Disease, Gene, Listed LPM Contreras as of 4/15/2015, NIEMANN-PICK, Post LPM Assignment Set 20150420, Pre LPM working set 20150418, THERAPY, treatment, TYPE, VEXXXX, VHXXXX, viral, WJXXXX, XEXXXX
Category(s): Collaboration Sought > Collaboration, TherapeuticArea > Endocrinology, Application > Therapeutics, TherapeuticArea > Neurology