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10.1194/jlr.R047167 http://scihub22266oqcxt.onion/10.1194/jlr.R047167 C4076080!4076080!24534703     free     free     free Deprecated: Implicit conversion from float 209.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 209.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 209.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 209.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 209.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 209.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 243.2 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534       J+Lipid+Res 2014 ; 55 (7): 1215-25 Nephropedia Template TP {{tp|p=24534703|t=2014. The development and use of small molecule inhibitors of glycosphingolipid metabolism for lysosomal storage diseases |pdf=|usr=}}{{24534703}} gab.com Text The development and use of small molecule inhibitors of glycosphingolipid metabolism for lysosomal storage diseases JO: J Lipid Res http://www.kidney.de/pget.php?&tw=1&pmid=24534703 #FOAvip Glycosphingolipid (GSL) storage diseases have been the focus of efforts to develop small molecule therapeutics from design, experimental proof of concept studies, and clinical trials. Two primary alternative strategies that have been pursued include pharmacological chaperones and GSL synthase inhibitors. There are theoretical advantages and disadvantages to each of these approaches. Pharmacological chaperones are specific for an individual glycoside hydrolase and for the specific mutation present, but no candidate chaperone has been demonstrated to be effective for all mutations leading to a given disorder. Synthase inhibitors target single enzymes such as glucosylceramide synthase and inhibit the formation of multiple GSLs. A glycolipid synthase inhibitor could potentially be used to treat multiple diseases, but at the risk of lowering nontargeted cellular GSLs that are important for normal health. The basis for these strategies and specific examples of compounds that have led to clinical trials is the focus of this review. Twit Text FOAVip The development and use of small molecule inhibitors of glycosphingolipid metabolism for lysosomal storage diseases ????J Lipid Res http://www.kidney.de/pget.php?&tw=1&pmid=24534703 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=24534703 #FOAvip English Wikipedia <ref>{{Cite pmid|24534703}}</ref> The development and use of small molecule inhibitors of glycosphingolipid metabolism for lysosomal storage diseases #MMPMID24534703 Shayman JA; Larsen SD J Lipid Res 2014[Jul]; 55 (7): 1215-25 PMID24534703show ga Glycosphingolipid (GSL) storage diseases have been the focus of efforts to develop small molecule therapeutics from design, experimental proof of concept studies, and clinical trials. Two primary alternative strategies that have been pursued include pharmacological chaperones and GSL synthase inhibitors. There are theoretical advantages and disadvantages to each of these approaches. Pharmacological chaperones are specific for an individual glycoside hydrolase and for the specific mutation present, but no candidate chaperone has been demonstrated to be effective for all mutations leading to a given disorder. Synthase inhibitors target single enzymes such as glucosylceramide synthase and inhibit the formation of multiple GSLs. A glycolipid synthase inhibitor could potentially be used to treat multiple diseases, but at the risk of lowering nontargeted cellular GSLs that are important for normal health. The basis for these strategies and specific examples of compounds that have led to clinical trials is the focus of this review. äThe development and use of small molecule inhibitors of glycosphingoli(epmc).pdf DeepDyve Pubget Overpricing