Conformational inhibitors of protein aggregation.

Fernández Ramírez, María Del Carmen; Afrin, Shumaila; Saelices, Lorena

Fernández Ramírez, María Del Carmen; Afrin, Shumaila; Saelices, Lorena.

Afiliación

Fernández Ramírez MDC; Center for Alzheimer's and Neurodegenerative Diseases, Department of Biophysics, Peter O'Donnell Jr Brain Institute, University of Texas Southwestern Medical Center (UTSW), Dallas, TX, USA. Electronic address: https://twitter.com/FernandezR_MC.
Afrin S; Center for Alzheimer's and Neurodegenerative Diseases, Department of Biophysics, Peter O'Donnell Jr Brain Institute, University of Texas Southwestern Medical Center (UTSW), Dallas, TX, USA. Electronic address: https://twitter.com/Shumyla44.
Saelices L; Center for Alzheimer's and Neurodegenerative Diseases, Department of Biophysics, Peter O'Donnell Jr Brain Institute, University of Texas Southwestern Medical Center (UTSW), Dallas, TX, USA. Electronic address: Lorena.SaelicesGomez@UTSouthwestern.edu.

Curr Opin Struct Biol ; 83: 102700, 2023 Dec.

Article en En | MEDLINE | ID: mdl-37717490

RESUMEN

Amyloidoses are fatal conditions associated with the aggregation of proteins into amyloid fibrils that deposit systemically and/or locally. Possibly because the causal mechanism of protein aggregation and deposition is not fully understood, this group of diseases remains uncurable. Advances in structural biology, such as the use of nuclear magnetic resonance and cryo-electron microscopy, have enabled the study of the structures and the conformational nature of the proteins whose aggregation is associated with the underlying pathogenesis of amyloidosis. As a result, the last years of research have translated into the development of directed therapeutic strategies that target the specific conformations of precursors, fibrils, and intermediary species. Current efforts include the use of small molecules, peptides, and antibodies. This review summarizes the recent progress in developing strategies that target specific protein conformations for the treatment of amyloidoses.

Asunto(s)

Amiloidosis; Agregado de Proteínas; Humanos; Microscopía por Crioelectrón; Amiloidosis/metabolismo; Amiloidosis/patología; Amiloide/química; Conformación Proteica

Palabras clave

Amyloid; Amyloidosis; Conformational inhibitors; Protein aggregation

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Agregado de Proteínas / Amiloidosis Límite: Humans Idioma: En Revista: Curr Opin Struct Biol Asunto de la revista: BIOLOGIA MOLECULAR Año: 2023 Tipo del documento: Article Pais de publicación: Reino Unido

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