Back to search

Article

Lowering mutant huntingtin by small molecules relieves Huntington’s disease symptoms and progression

2023-11-21

Abstract excerpt

Huntington’s disease (HD) is an incurable inherited disorder caused by repeat expansion in the huntingtin gene ( Htt ). The mutant protein causes neuronal degeneration leading to severe motor and psychological abnormalities. Selective downregulation of the mutant Htt expression is considered the leading therapeutic approach for HD. We report the identification of novel small molecule inhibitors of Spt5-Pol II, S...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work
9f54da60-c3b7-5c57-9631-43e543d33595
DOI
10.1101/2023.11.21.568060
Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest
Interactive article relationship graphSelect a related publication card to move it into the centre and load its closest explainable connections. Solid lines are source-backed structured connections. Dashed lines are semantic discovery signals and are not scientific evidence.
Lowering mutant huntingtin by small molecules relieves Huntington’s disease symptoms and progressionDOI 10.1101/2023.11.21.568060
Lowering mutant huntingtin by small molecules relieves Huntington's disease symptoms and progression.Semantic proximity
Huntington's disease: progress toward effective disease-modifying treatments and a cure.Semantic proximity
The Emerging Landscape of Small-Molecule Therapeutics for the Treatment of Huntington’s DiseaseSemantic proximity
Therapeutic Advances for Huntington’s DiseaseSemantic proximity
Delivering a disease-modifying treatment for Huntington's disease.Semantic proximity
An insight into allele-selective approaches to lowering mutant huntingtin protein for Huntington's disease treatment.Semantic proximity
Huntingtin-lowering strategies for Huntington's disease.Semantic proximity
An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusionSemantic proximity
Therapeutic strategies for Huntington's diseaseSemantic proximity
Therapeutics Advancement for Huntington DiseaseSemantic proximity
Emerging therapies in Huntington’s diseaseSemantic proximity
Gene therapy in mouse models of huntington disease.Semantic proximity
Select a neighboring publication to make it the new centre.