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ProteomicsProteomic changes upon huntingtin lowering in human cortical neurons
Despite growing descriptions of wild-type Huntingtin's (wt-HTT) roles in both adult brain function and, more recently, development, several clinical trials are exploring HTT-lowering approaches that target both wt-HTT and the mutant isoforms (mut-HTT) responsible for Huntington's disease (HD). This ...
ORGANISM(S): Homo sapiens (Human) 
2025-03-25 | PXD053150 | Pride
Neuronal network  htt lowering  huntington disease  huntingtin  pluripotent stem cell  synapse  proteome  nanolc-msms  label free quantification of proteins 
UnknownPreclinical evaluation of stereopure antisense oligonucleotides for allele-selective lowering of mutant HTT.
Not available
S-EPMC11255113 | biostudies-literature
UnknownSmall molecule splicing modifiers with systemic HTT-lowering activity.
Not available
S-EPMC8674292 | biostudies-literature
ProteomicsStriatum Proteomics from LacQ140 mutant Huntingtin lowering mouse model
Lowering of mutant Huntingtin (mHtt) transcript and protein levels is considered a therapeutic strategy for people with Huntington’s disease (HD). However, the effects of mHtt lowering are incompletely understood. In this study, the proteomic changes in striatum upon Htt lowering were analyzed, usin...
ORGANISM(S): Mus musculus (Mouse) 
2025-04-11 | PXD054495 | Pride
Striata Mouse Huntington’s disease Lc-ms/ms 
UnknownMolecular and imaging biomarker responses to brain mutant HTT lowering in a mouse model of Huntington disease.
Not available
S-EPMC12489819 | biostudies-literature
GenomicsSmall molecule activation of a pseudoexon triggers huntingtin-lowering (AmpliSeq)
Small molecule activation of a pseudoexon triggers huntingtin-lowering (AmpliSeq)
PRJNA683141 | ENA
GenomicsTranscriptional profiling of striatum and cortex from a LacO-Q140 inducible mouse model of Huntington’s disease with early and late mutant HTT lowering
Transcriptional profiling of striatum and cortex from a LacO-Q140 inducible mouse model of Huntington’s disease with early and late mutant HTT lowering
PRJNA657116 | ENA
GenomicsSmall molecule activation of a pseudoexon triggers huntingtin-lowering (RNA-Seq)
Small molecule activation of a pseudoexon triggers huntingtin-lowering (RNA-Seq)
PRJNA683140 | ENA
UnknownHuR stabilizes HTT mRNA via interacting with its exon 11 in a mutant HTT-dependent manner.
Not available
S-EPMC7237150 | biostudies-literature
TranscriptomicsSmall molecule activation of a pseudoexon triggers huntingtin-lowering (AmpliSeq)
Huntington’s disease (HD) is a hereditary neurodegenerative disorder caused by abnormal expansion of cytosine-adenine-guanine (CAG) trinucleotide repeats in the huntingtin gene (HTT). The resultant mutant protein is ubiquitously expressed and drives pathogenesis of HD through a toxic gain-of-functio...
ORGANISM(S): Homo sapiens 
2021-07-01 | GSE162812 | GEO
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