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Early whole-body mutant huntingtin lowering averts changes in proteins and lipids important for synapse function and white matter maintenance in the LacQ140 mouse model

BACKGROUND: Expansion of a triplet repeat tract in exon1 of the HTT gene causes Huntington’s disease (HD). The mutant HTT protein (mHTT) has numerous aberrant interactions with diverse, pleiomorphic effects. No disease modifying treatments exist but lowering mutant huntingtin (mHTT) by gene therapy...

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Detalles Bibliográficos
Autores principales: Shing, Kai, Sapp, Ellen, Boudi, Adel, Liu, Sophia, Seeley, Connor, Marchionini, Deanna, DiFiglia, Marian, Kegel-Gleason, Kimberly B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cold Spring Harbor Laboratory 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9900921/
https://www.ncbi.nlm.nih.gov/pubmed/36747614
http://dx.doi.org/10.1101/2023.01.26.525697