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Int J Mol Sci ; 21(9)2020 May 09.
Artículo en Inglés | MEDLINE | ID: mdl-32397443

RESUMEN

Iminosugars are sugar analogues endowed with a high pharmacological potential. The wide range of biological activities exhibited by these glycomimetics associated with their excellent drug profile make them attractive therapeutic candidates for several medical interventions. The ability of iminosugars to act as inhibitors or enhancers of carbohydrate-processing enzymes suggests their potential use as therapeutics for the treatment of cystic fibrosis (CF). Herein we review the most relevant advances in the field, paying attention to both the chemical synthesis of the iminosugars and their biological evaluations, resulting from in vitro and in vivo assays. Starting from the example of the marketed drug NBDNJ (N-butyl deoxynojirimycin), a variety of iminosugars have exhibited the capacity to rescue the trafficking of F508del-CFTR (deletion of F508 residue in the CF transmembrane conductance regulator), either alone or in combination with other correctors. Interesting results have also been obtained when iminosugars were considered as anti-inflammatory agents in CF lung disease. The data herein reported demonstrate that iminosugars hold considerable potential to be applied for both therapeutic purposes.


Asunto(s)
Fibrosis Quística/tratamiento farmacológico , Compuestos Heterocíclicos con 1 Anillo/uso terapéutico , 1-Desoxinojirimicina/análogos & derivados , 1-Desoxinojirimicina/química , 1-Desoxinojirimicina/uso terapéutico , Antiinflamatorios/química , Antiinflamatorios/uso terapéutico , Regulador de Conductancia de Transmembrana de Fibrosis Quística/genética , Regulador de Conductancia de Transmembrana de Fibrosis Quística/metabolismo , Glicósido Hidrolasas/antagonistas & inhibidores , Glicosiltransferasas/antagonistas & inhibidores , Compuestos Heterocíclicos con 1 Anillo/síntesis química , Compuestos Heterocíclicos con 1 Anillo/química , Humanos , Iminopiranosas/química , Iminopiranosas/uso terapéutico , Inflamación , Estructura Molecular , Mutación , Eliminación de Secuencia , Tartratos/química , Tartratos/uso terapéutico
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