A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols
[Display omitted] •A short and high yielding route enables access to N-substituted amino(hydroxymethyl)pentanetriols.•Final products are potent inhibitors of a panel of β-galactosidases.•New compounds are efficient pharmacological chaperones for GM1-gangliosidosis-related human lysosomal β-galactosi...
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| Veröffentlicht in: | Bioorganic & medicinal chemistry letters 2017-08, Vol.27 (15), p.3431-3435 |
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| container_end_page | 3435 |
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| container_issue | 15 |
| container_start_page | 3431 |
| container_title | Bioorganic & medicinal chemistry letters |
| container_volume | 27 |
| creator | Schalli, Michael Weber, Patrick Tysoe, Christina Pabst, Bettina M. Thonhofer, Martin Paschke, Eduard Stütz, Arnold E. Tschernutter, Marion Windischhofer, Werner Withers, Stephen G. |
| description | [Display omitted]
•A short and high yielding route enables access to N-substituted amino(hydroxymethyl)pentanetriols.•Final products are potent inhibitors of a panel of β-galactosidases.•New compounds are efficient pharmacological chaperones for GM1-gangliosidosis-related human lysosomal β-galactosidase.
N-Functionalized amino(hydroxymethyl)cyclopentanetriols are potent inhibitors of β-d-galactosidases and, for the first time, could be shown to act as pharmacological chaperones for GM1-gangliosidosis-associated lysosomal acid β-galactosidase thus representing a new structural type of pharmacological chaperones for this lysosomal storage disease. |
| doi_str_mv | 10.1016/j.bmcl.2017.05.086 |
| format | Article |
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•A short and high yielding route enables access to N-substituted amino(hydroxymethyl)pentanetriols.•Final products are potent inhibitors of a panel of β-galactosidases.•New compounds are efficient pharmacological chaperones for GM1-gangliosidosis-related human lysosomal β-galactosidase.
N-Functionalized amino(hydroxymethyl)cyclopentanetriols are potent inhibitors of β-d-galactosidases and, for the first time, could be shown to act as pharmacological chaperones for GM1-gangliosidosis-associated lysosomal acid β-galactosidase thus representing a new structural type of pharmacological chaperones for this lysosomal storage disease.</description><identifier>ISSN: 0960-894X</identifier><identifier>EISSN: 1464-3405</identifier><identifier>DOI: 10.1016/j.bmcl.2017.05.086</identifier><language>eng</language><publisher>Elsevier Ltd</publisher><subject>Aminocyclopentane ; Carbafuranose ; Galactosidase inhibitor ; GM1-Gangliosidosis ; Pharmacological chaperone</subject><ispartof>Bioorganic & medicinal chemistry letters, 2017-08, Vol.27 (15), p.3431-3435</ispartof><rights>2017 Elsevier Ltd</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://dx.doi.org/10.1016/j.bmcl.2017.05.086$$EHTML$$P50$$Gelsevier$$H</linktohtml><link.rule.ids>314,780,784,3550,27924,27925,45995</link.rule.ids></links><search><creatorcontrib>Schalli, Michael</creatorcontrib><creatorcontrib>Weber, Patrick</creatorcontrib><creatorcontrib>Tysoe, Christina</creatorcontrib><creatorcontrib>Pabst, Bettina M.</creatorcontrib><creatorcontrib>Thonhofer, Martin</creatorcontrib><creatorcontrib>Paschke, Eduard</creatorcontrib><creatorcontrib>Stütz, Arnold E.</creatorcontrib><creatorcontrib>Tschernutter, Marion</creatorcontrib><creatorcontrib>Windischhofer, Werner</creatorcontrib><creatorcontrib>Withers, Stephen G.</creatorcontrib><title>A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols</title><title>Bioorganic & medicinal chemistry letters</title><description>[Display omitted]
•A short and high yielding route enables access to N-substituted amino(hydroxymethyl)pentanetriols.•Final products are potent inhibitors of a panel of β-galactosidases.•New compounds are efficient pharmacological chaperones for GM1-gangliosidosis-related human lysosomal β-galactosidase.
N-Functionalized amino(hydroxymethyl)cyclopentanetriols are potent inhibitors of β-d-galactosidases and, for the first time, could be shown to act as pharmacological chaperones for GM1-gangliosidosis-associated lysosomal acid β-galactosidase thus representing a new structural type of pharmacological chaperones for this lysosomal storage disease.</description><subject>Aminocyclopentane</subject><subject>Carbafuranose</subject><subject>Galactosidase inhibitor</subject><subject>GM1-Gangliosidosis</subject><subject>Pharmacological chaperone</subject><issn>0960-894X</issn><issn>1464-3405</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2017</creationdate><recordtype>article</recordtype><recordid>eNotkTGO1TAURSMEEp-BDVC5pHF4TmwnQTSjEQxIAxSARGfZzsuPv5w42A6Q5bAFFsKaSDQUV7c57-lKpyieMygZMPnyUprJ-rIC1pQgSmjlg-LEuOS05iAeFifoJNC2498eF09SugAwDpyfit_XZMafJG8LkjCQZdRx0jb4cHZWe2JHvWAMM5IhRHL7gdGzns_eheT6PYlE9DpjT8Z10jPxWwopTPvh3z876bXNB6kTviIf6efVpOzyevCC6snNgTI6bn0Mv7YJ87h5ajfrw4Jz1jPm6IJPT4tHg_YJn_3vq-Lr2zdfbt7Ru0-372-u7ygyITPltuoGxgAkN9hW0jSsr1vgAxguKtnYth86jQakNY0wWGvDLO8Nq3Tbd0LUV8WL-79LDN9XTFlNLln0fl8S1qRYBy2vWt5UO_r6HsV9zw-HUSXrcLbYu4g2qz44xUAdZtRFHWbUYUaBULuZ-h_yGIi3</recordid><startdate>20170801</startdate><enddate>20170801</enddate><creator>Schalli, Michael</creator><creator>Weber, Patrick</creator><creator>Tysoe, Christina</creator><creator>Pabst, Bettina M.</creator><creator>Thonhofer, Martin</creator><creator>Paschke, Eduard</creator><creator>Stütz, Arnold E.</creator><creator>Tschernutter, Marion</creator><creator>Windischhofer, Werner</creator><creator>Withers, Stephen G.</creator><general>Elsevier Ltd</general><scope>7X8</scope></search><sort><creationdate>20170801</creationdate><title>A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols</title><author>Schalli, Michael ; Weber, Patrick ; Tysoe, Christina ; Pabst, Bettina M. ; Thonhofer, Martin ; Paschke, Eduard ; Stütz, Arnold E. ; Tschernutter, Marion ; Windischhofer, Werner ; Withers, Stephen G.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-e156t-4c29f110064be826b71d3804f0b45267c8df9aeb06cb75be3ab1c4db12a8d9553</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2017</creationdate><topic>Aminocyclopentane</topic><topic>Carbafuranose</topic><topic>Galactosidase inhibitor</topic><topic>GM1-Gangliosidosis</topic><topic>Pharmacological chaperone</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Schalli, Michael</creatorcontrib><creatorcontrib>Weber, Patrick</creatorcontrib><creatorcontrib>Tysoe, Christina</creatorcontrib><creatorcontrib>Pabst, Bettina M.</creatorcontrib><creatorcontrib>Thonhofer, Martin</creatorcontrib><creatorcontrib>Paschke, Eduard</creatorcontrib><creatorcontrib>Stütz, Arnold E.</creatorcontrib><creatorcontrib>Tschernutter, Marion</creatorcontrib><creatorcontrib>Windischhofer, Werner</creatorcontrib><creatorcontrib>Withers, Stephen G.</creatorcontrib><collection>MEDLINE - Academic</collection><jtitle>Bioorganic & medicinal chemistry letters</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Schalli, Michael</au><au>Weber, Patrick</au><au>Tysoe, Christina</au><au>Pabst, Bettina M.</au><au>Thonhofer, Martin</au><au>Paschke, Eduard</au><au>Stütz, Arnold E.</au><au>Tschernutter, Marion</au><au>Windischhofer, Werner</au><au>Withers, Stephen G.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols</atitle><jtitle>Bioorganic & medicinal chemistry letters</jtitle><date>2017-08-01</date><risdate>2017</risdate><volume>27</volume><issue>15</issue><spage>3431</spage><epage>3435</epage><pages>3431-3435</pages><issn>0960-894X</issn><eissn>1464-3405</eissn><abstract>[Display omitted]
•A short and high yielding route enables access to N-substituted amino(hydroxymethyl)pentanetriols.•Final products are potent inhibitors of a panel of β-galactosidases.•New compounds are efficient pharmacological chaperones for GM1-gangliosidosis-related human lysosomal β-galactosidase.
N-Functionalized amino(hydroxymethyl)cyclopentanetriols are potent inhibitors of β-d-galactosidases and, for the first time, could be shown to act as pharmacological chaperones for GM1-gangliosidosis-associated lysosomal acid β-galactosidase thus representing a new structural type of pharmacological chaperones for this lysosomal storage disease.</abstract><pub>Elsevier Ltd</pub><doi>10.1016/j.bmcl.2017.05.086</doi><tpages>5</tpages></addata></record> |
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| ispartof | Bioorganic & medicinal chemistry letters, 2017-08, Vol.27 (15), p.3431-3435 |
| issn | 0960-894X 1464-3405 |
| language | eng |
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| source | Access via ScienceDirect (Elsevier) |
| subjects | Aminocyclopentane Carbafuranose Galactosidase inhibitor GM1-Gangliosidosis Pharmacological chaperone |
| title | A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols |
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