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 in intracellular cholesterol trafficking. Mutations of NPC1 cause intracellular accumulation of unesterified cholesterol in late endosomal/lysosomal structures and marked accumulation of glycosphingolipids, especially in neuronal tissue.

Method of Detecting Circulating Cell-Free HPV 6 and 11 DNA in Patients Afflicted With Diseases Caused by Chronic HPV 6 or 11 Infection and Use Thereof

Summary:

The National Cancer Institute (NCI) and Frederick National Laboratory for Cancer Research (FNLCR) seek research co-development partners and/or licensees for commercial development of a novel liquid biopsy diagnostic for non-invasive detection of cell-free HPV 6 and 11 DNA for recurrent respiratory papillomatosis (RRP).