Naphthyridine-Azaquinolone reverses Huntington’s DNA repeats in mouse model

Naphthyridine-Azaquinolone reverses Huntington’s DNA repeats in mouse model

Publication date: Feb 29, 2020

The study published in Nature Genetics details how international collaborators from The Hospital for Sick Children (SickKids) in Canada and research teams from Japan’s Osaka University were able to reverse repeat mutation length in the brains of a mouse model with Huntington’s disease.

In Huntington’s and similar conditions, the more repeat sequences (ie, the greater the repeat mutation length), the more severe the symptoms and the earlier the onset and the faster the progression of the disease.

Since longer expansions over time are directly associated with more severe disease, our findings offer hope for the ability to delay the onset of Huntington’s and slow its progression,” said study principal investigator Dr Christopher Pearson, SickKids Senior Scientist in Genetics & Genome Biology.

Concepts Keywords
ATE Characteristic neurodegenerative diseases
Brain Genetics
Canada RTT
DNA Trinucleotide repeat disorders
FAT Autosomal dominant disorders
Huntington Branches of biology
Kick Start Contractions disease
Murine Directly severe disease
Mutant Medicine
Mutation Myotonic dystrophy
Myotonic Dystrophy Huntington’s disease
Nature Mutation
Neurodegenerative Diseases CAT
Principal Investigator
Small Molecule
The Collaborators


Type Source Name
disease MESH DNA repeat expansions
disease MESH neurodegenerative diseases
disease MESH development
disease MESH myotonic dystrophy


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