Chaperone Nanobodies Protect Gelsolin Against MT1-MMP Degradation and Alleviate Amyloid Burden in the Gelsolin Amyloidosis Mouse Model

Gelsolin amyloidosis is an autosomal dominant incurable disease caused by a point mutation in the GSN gene (G654A/T), specifically affecting secreted plasma gelsolin. Incorrect folding of the mutant (D187N/Y) second gelsolin domain leads to a pathological proteolytic cascade. D187N/Y gelsolin is fir...

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Publicado en:Mol Ther
Main Authors: Van Overbeke, Wouter, Verhelle, Adriaan, Everaert, Inge, Zwaenepoel, Olivier, Vandekerckhove, Joël, Cuvelier, Claude, Derave, Wim, Gettemans, Jan
Formato: Artigo
Idioma:Inglês
Publicado: Nature Publishing Group 2014
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Original Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC4428403/
https://ncbi.nlm.nih.gov/pubmed/25023329
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2014.132
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