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Invention E-050-2012/0

Cyclodextrins as Therapeutics for Lysosomal Storage Disorders

Lead Inventor: Wei Zheng (NCATS)
Inventors: Juan Marugan (NCATS), John McKew (NCATS), Manju Swaroop (NHGRI), Miao Xu (NHGRI)
Ref. No.: E-050-2012/0

Abstract: Cyclodextrins, alone or in combination with other agents (e.g., vitamin E), as therapeutics for the treatment of lysosomal storage disorders caused by the accumulation of non-cholesterol lipids.

Publications:

δ-Tocopherol Reduces Lipid Accumulation in Niemann-Pick Type C1 and Wolman Cholesterol Storage Disorders
Journal of Biological Chemistry • Nov. 16, 2012 • Therapeutics for Rare and Neglected Diseases

Evaluation of Cholesterol Reduction Activity of Methyl-β-Cyclodextrin Using Differentiated Human Neurons and AstrocytesJournal of Biomolecular Screening • October 2012 • Therapeutics for Rare and Neglected Diseases

Last updated: 07-21-2016
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