Methods for treating mucopolysaccharidosis

a technology of mucopolysaccharidosis and compositions, applied in the field of compositions and methods for treating mucopolysaccharidosis, can solve the problems of insufficient production of enzymes required to degrade gags, defective enzymes that do not function properly, and undegraded or partially degraded gags, etc., to achieve the effect of increasing enzyme expression and increasing idua activity

Inactive Publication Date: 2019-03-14
UNIV OF MIAMI
View PDF0 Cites 0 Cited by
  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Benefits of technology

[0006]The present invention provides compositions and methods for treating mucopolysaccharidosis (MPS). The methods of the invention comprise administering to a subject in need thereof a therapeutically effective amount of a stilbene or stilbenoid compound, such as one or more hydroxylated or carboxylated stilbenoids, such as resveratrol, piceatannol, or 4,4′-stilbenedicarboxylic acid. In various embodiments, the invention provides methods for treating MPS I-H, MPS I-S, MPS I-H/S, MPS II, MPS III A, MPS II

Problems solved by technology

MPS patients either do not produce enough of the enzymes required to degrade GAGs or produce defective enzymes that do not function properly.
As a result, undegraded or partially degraded GAGs accumulate and interfere with the normal function of cells, tissues, and organs, and affect the normal growth and development of individuals.
Hurler syndrome i

Method used

the structure of the environmentally friendly knitted fabric provided by the present invention; figure 2 Flow chart of the yarn wrapping machine for environmentally friendly knitted fabrics and storage devices; image 3 Is the parameter map of the yarn covering machine
View more

Image

Smart Image Click on the blue labels to locate them in the text.
Viewing Examples
Smart Image
  • Methods for treating mucopolysaccharidosis
  • Methods for treating mucopolysaccharidosis
  • Methods for treating mucopolysaccharidosis

Examples

Experimental program
Comparison scheme
Effect test

example 1

Administration of Resveratrol Restores IDUA Expression and / or Activity

[0071]A cell culture experiment was performed in MPS-I patient-derived fibroblast cells. Cells were cultured overnight in a 384-well plate, at a concentration of 20,000 cells per well in advanced DMEM media (Life Technologies) supplemented with 15% FBS (Life technologies), 1× Glutamax (Life Technologies) and beta-mercaptoethanol (1:125000 dilution). The next day, cells were treated with the indicated compound with final concentrations including, but not limited to: 100 μM, 30 μM, 10 μM, 3.3 μM, 1.1 μM, 0.37 μM and 0.12 μM depending on experiment. Treated cells were then incubated for 48 hours at 37° C., 5% CO2, 95% humidity. Cells were then washed with HBSS buffer, and 25 μM of the fluorogenic substrate 4-Methylumbelliferyl α-L-iduronide (free acid) (Santa Cruz Biotechnology) were added in the presence of 0.2M CHNaO2, 0.1% BSA, and 0.1% Triton X. After the addition of the 4-Methylumbelliferyl α-L-iduronide substra...

the structure of the environmentally friendly knitted fabric provided by the present invention; figure 2 Flow chart of the yarn wrapping machine for environmentally friendly knitted fabrics and storage devices; image 3 Is the parameter map of the yarn covering machine
Login to view more

PUM

PropertyMeasurementUnit
Massaaaaaaaaaa
Massaaaaaaaaaa
Login to view more

Abstract

The present invention provides methods and compositions for the treatment of mucopolysaccharidoses (MPS).

Description

CROSS-REFERENCE TO RELATED APPLICATION[0001]This application is a National Stage Application of International Patent Application No. PCT / US2016 / 041564, filed Jul. 8, 2016, which claims the priority benefit of U.S. Provisional Patent Application No. 62 / 190,935, filed Jul. 10, 2015, the disclosure of which is incorporated herein by reference in its entirety.FIELD OF THE INVENTION[0002]The present invention relates to compositions and methods for treating mucopolysaccharidosis (MPS).BACKGROUND[0003]Mucopolysaccharidosis (MPS) constitutes a class of lysosomal storage disorders characterized by the excessive accumulation of glycosaminoglycans (GAGs) within the lysosomes of various tissues. MPS patients either do not produce enough of the enzymes required to degrade GAGs or produce defective enzymes that do not function properly. As a result, undegraded or partially degraded GAGs accumulate and interfere with the normal function of cells, tissues, and organs, and affect the normal growth ...

Claims

the structure of the environmentally friendly knitted fabric provided by the present invention; figure 2 Flow chart of the yarn wrapping machine for environmentally friendly knitted fabrics and storage devices; image 3 Is the parameter map of the yarn covering machine
Login to view more

Application Information

Patent Timeline
no application Login to view more
IPC IPC(8): A61K31/05A61K9/00A61P3/00
CPCA61K31/05A61K9/0019A61K9/0053A61P3/00A61K45/06A61K31/03A61K31/09A61K31/166A61K31/185A61K31/194A61K31/235A61K31/36A61K31/423A61K31/69A61K31/7004
Inventor BROTHERS, SHAUNWAHLESTEDT, CLAESVOLMAR, CLAUDE HENRY
Owner UNIV OF MIAMI
Who we serve
  • R&D Engineer
  • R&D Manager
  • IP Professional
Why Eureka
  • Industry Leading Data Capabilities
  • Powerful AI technology
  • Patent DNA Extraction
Social media
Try Eureka
PatSnap group products

Browse by: Latest US Patents, China's latest patents, Technical Efficacy Thesaurus, Application Domain, Technology Topic.

© 2024 PatSnap. All rights reserved.Legal|Privacy policy|Modern Slavery Act Transparency Statement|Sitemap