Enzyme enhancement therapy through non-competitive pharmacological chaperones
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Cita com:
hdl:2117/24997
Tipus de documentText en actes de congrés
Data publicació2014
Condicions d'accésAccés obert
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Abstract
Most Pharmacological chaperones (PC’s) described until now are
substrate analogues which bind to the active site of the target protein. C
ons
e-
quently, such PC’s also inhibit the target protein at higher concentrations thus
rendering a narrow therapeutic window and have poor drug-like properties.
Through our proprietary technology platform SEE-Tx™, we identify a new
generation of non-substrate competitive pharmacological chaperones which p
o-
tentially offer a much broader therapeutic window. What’s more, such co
m-
pounds are not substrate analogues, thus presenting much better drug-like pro
p-
erties, particularly for indications with CNS involvement. Here we present our
methodology to identify non-competitive pharmacological chaperones applied
to the enzyme beta-galactosidase, whose deficiency is related with GM1
Gangliosidosis and Morquio B.
Descripció
Pharmacological chaperones, Chemical chaperones
,
Enzyme e
n-
hancement
therapy, GM1, Gangliosidosis, Morqui B, Lysosomal Storage Disease,
Lysosomal Storage Disorders
CitacióAymami, J. [et al.]. Enzyme enhancement therapy through non-competitive pharmacological chaperones. A: IWBBIO 2014. "Proceedings IWBBIO 2014". 2014, p. 390-395.
Versió de l'editorhttp://iwbbio.ugr.es/2014/papers/IWBBIO_2014_paper_45.pdf
Fitxers | Descripció | Mida | Format | Visualitza |
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IWBBIO_2014_paper_45.pdf | proceedings IWBBIO 2014 | 844,1Kb | Visualitza/Obre |