Hydroxy piperidine derivatives to treat gaucher disease
The present invention provides novel hydroxy piperidine (HP) derivatives having (i) a positive charge at the position corresponding to the anomeric position of a pyranose ring; (ii) a short, flexible linker emanating from the corresponding position of the ring oxygen in a pyranose; and (iii) a lipop...
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| creator | FAN JIAN-QIANG,ZHU XIAOXIANG,SHETH KAMLESH |
| description | The present invention provides novel hydroxy piperidine (HP) derivatives having (i) a positive charge at the position corresponding to the anomeric position of a pyranose ring; (ii) a short, flexible linker emanating from the corresponding position of the ring oxygen in a pyranose; and (iii) a lipophilic moiety connected to the linker and pharmaceutically acceptable salts thereof. The linker can be absent if the lipophilic moiety corresponds to a hydrocarbon chain with a linear length of 6 or more carbons. The present invention further provides a method for treating individuals having Gaucher disease by administering the novel HP derivative as "active-site specific chaperones" for the mutant glucocerebrosidase associated with the disease. |
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The linker can be absent if the lipophilic moiety corresponds to a hydrocarbon chain with a linear length of 6 or more carbons. 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The linker can be absent if the lipophilic moiety corresponds to a hydrocarbon chain with a linear length of 6 or more carbons. The present invention further provides a method for treating individuals having Gaucher disease by administering the novel HP derivative as "active-site specific chaperones" for the mutant glucocerebrosidase associated with the disease.</description><subject>CHEMISTRY</subject><subject>HETEROCYCLIC COMPOUNDS</subject><subject>HUMAN NECESSITIES</subject><subject>HYGIENE</subject><subject>MEDICAL OR VETERINARY SCIENCE</subject><subject>METALLURGY</subject><subject>ORGANIC CHEMISTRY</subject><subject>PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</subject><subject>SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS</subject><fulltext>true</fulltext><rsrctype>patent</rsrctype><creationdate>2007</creationdate><recordtype>patent</recordtype><sourceid>EVB</sourceid><recordid>eNrjZDD3qEwpyq-oVCjILEgtykzJzEtVSAEyyhJLMstSixVK8hVKilITSxTSE0uTM1KLFFIyi1MTi1N5GFjTEnOKU3mhNDeDvJtriLOHbmpBfnxqcUFicmpeakm8s5-hpYGZoZmRozFhFQBeny2S</recordid><startdate>20070131</startdate><enddate>20070131</enddate><creator>FAN JIAN-QIANG,ZHU XIAOXIANG,SHETH KAMLESH</creator><scope>EVB</scope></search><sort><creationdate>20070131</creationdate><title>Hydroxy piperidine derivatives to treat gaucher disease</title><author>FAN JIAN-QIANG,ZHU XIAOXIANG,SHETH KAMLESH</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-epo_espacenet_CN1906162A3</frbrgroupid><rsrctype>patents</rsrctype><prefilter>patents</prefilter><language>eng</language><creationdate>2007</creationdate><topic>CHEMISTRY</topic><topic>HETEROCYCLIC COMPOUNDS</topic><topic>HUMAN NECESSITIES</topic><topic>HYGIENE</topic><topic>MEDICAL OR VETERINARY SCIENCE</topic><topic>METALLURGY</topic><topic>ORGANIC CHEMISTRY</topic><topic>PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</topic><topic>SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS</topic><toplevel>online_resources</toplevel><creatorcontrib>FAN JIAN-QIANG,ZHU XIAOXIANG,SHETH KAMLESH</creatorcontrib><collection>esp@cenet</collection></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext_linktorsrc</fulltext></delivery><addata><au>FAN JIAN-QIANG,ZHU XIAOXIANG,SHETH KAMLESH</au><format>patent</format><genre>patent</genre><ristype>GEN</ristype><title>Hydroxy piperidine derivatives to treat gaucher disease</title><date>2007-01-31</date><risdate>2007</risdate><abstract>The present invention provides novel hydroxy piperidine (HP) derivatives having (i) a positive charge at the position corresponding to the anomeric position of a pyranose ring; (ii) a short, flexible linker emanating from the corresponding position of the ring oxygen in a pyranose; and (iii) a lipophilic moiety connected to the linker and pharmaceutically acceptable salts thereof. The linker can be absent if the lipophilic moiety corresponds to a hydrocarbon chain with a linear length of 6 or more carbons. The present invention further provides a method for treating individuals having Gaucher disease by administering the novel HP derivative as "active-site specific chaperones" for the mutant glucocerebrosidase associated with the disease.</abstract><oa>free_for_read</oa></addata></record> |
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| subjects | CHEMISTRY HETEROCYCLIC COMPOUNDS HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY ORGANIC CHEMISTRY PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS |
| title | Hydroxy piperidine derivatives to treat gaucher disease |
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