Target

HTT

Huntingtin

2open trials
7trials in total
23publications
0researchers

Diseases

The last 24 months

New trials, publications, preprints and grants on this target, month by month.

TrialsPublicationsPreprintsGrants

Publication
The Power of Two: Combined MSH3/HTT Silencing in Huntington's Disease.
15 Oct 2026· Movement disorders : official journal of the Movement Disorder Society· PUBMED
Publication
Loss of interruption in the HTT CAG repeat is associated with somatic expansion and loss of medium spiny neurons in Huntington's disease.
14 Sept 2026· Neuron· PUBMED
Publication
The HTT1a protein initiates HTT aggregation in a knock-in mouse model of Huntington's disease.
7 Sept 2026· Brain : a journal of neurology· PUBMED
Publication
Human-specific sequence features in HTT exon 1 promote toxic misprocessing via splicing factor SRSF7.
31 Jul 2026· Nature communications· PUBMED
Publication
Which HTT transcript to lower?
9 Jun 2026· Trends in pharmacological sciences· PUBMED
Publication
Ethnic Bridging of Votoplam, a HTT Gene Splicing Modifier, Using Pharmacokinetic Comparison Between Caucasian and Japanese Subjects.
1 May 2026· Clinical pharmacology in drug development· PUBMED
Publication
Treatment of Huntington's disease with a pan-HTT-targeting CRISPR nuclease.
24 Apr 2026· Molecular therapy : the journal of the American Society of Gene Therapy· PUBMED
Publication
Connecting HTT intermediate alleles and microRNA dysregulation to enhanced tauopathy in late-onset Alzheimer's disease.
20 Apr 2026· Alzheimer's research & therapy· PUBMED
Publication
Towards AI-driven prediction of HTT CAG size in super-expanded human spiny projection neurons from Huntington disease donors.
20 Apr 2026· Journal of Huntington's disease· PUBMED
Publication
Extensive transcriptomic changes in cellular and animal models of Huntington's disease depending on the length of CAG repeats in the exon 1 of the HTT gene.
1 Apr 2026· Biochemical and biophysical research communications· PUBMED
Publication
Huntingtin (HTT) interactome in regulation of DNA repair/remodeling and RNA processing pathways.
18 Mar 2026· Life science alliance· PUBMED
Publication
Silencing of human HTT by targeted CRISPR/dCas9-mediated epigenetic editing.
22 Jan 2026· Journal of Huntington's disease· PUBMED
Publication
Lifetime risk of cancer in carriers of intermediate alleles in the HTT gene.
16 Jan 2026· Scientific reports· PUBMED
Trial
Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease
6 Jan 2026· PHASE2_PHASE3 · RECRUITING· CTGOV
Publication
Localization of mutant huntingtin with HTT Exon1 P90 C-terminal neoepitope antibodies in relation to regional and neuronal vulnerability in forebrain in Q175 mice and human huntington's disease.
23 Dec 2025· Journal of Huntington's disease· PUBMED
Preprint
Treatment of Huntington's disease with a pan-HTT-targeting CRISPR nuclease.
22 Dec 2025· bioRxiv : the preprint server for biology· PUBMED
Publication
Sequence Variants in Small CAG Repeat Expansions of the HTT Gene and Disease Onset and Progression in Huntington Disease.
19 Dec 2025· Neurology· PUBMED
Publication
A human CAGinSTEM platform for decoding HTT repeats' somatic instability links CAG interruption to HD pathology in neurons.
11 Dec 2025· Cell reports· PUBMED
Publication
Effects of High- and Low-Fat Meals on the Bioavailability and Pharmacokinetics of Votoplam, a HTT Gene Splicing Modifier.
30 Nov 2025· Clinical pharmacology in drug development· PUBMED
Preprint
Sex- and Depression-specific Effects of Non-pathogenic CAG Repeats in HTT , ATXN3 and CACNA1A on Sleep.
27 Nov 2025· medRxiv : the preprint server for health sciences· PUBMED
Publication
Hyperkinesia and early-onset dementia in a female with co-occurring PSEN1 and HTT mutations: A case report.
3 Nov 2025· Journal of Alzheimer's disease reports· PUBMED
Publication
Intrastriatal Delivery of a Zinc Finger Protein Targeting the Mutant HTT Gene Allele Obviates Lipid Phenotypes in Brain and Plasma in Huntington's Disease Mice.
23 Jul 2025· Human gene therapy· PUBMED
Trial
Study of SKY-0515 for Safety, Efficacy, and Pharmacodynamics in Participants With Huntington's Disease
27 May 2025· PHASE2_PHASE3 · ACTIVE_NOT_RECRUITING· CTGOV

Who works on it

Researchers who published on this target. Leave out your own co-author circle to find people you do not know yet.

No published researcher profile on this target yet.

We look for a fixed list of target names in titles, keywords, intervention names and MeSH terms. A mention does not prove that the work is aimed at that target.

Report an error