Huntington's disease is a rare genetic, autosomal dominant, neurodegenerative disease, caused by CAG repeat expansions in the HTT gene, that is polymorphic on the healthy allele and contains more than ...
Germline CAG-repeat expansions mean that the children of individuals with Huntington’s disease would inherit CAG repeats that are longer than those in the previous generation, increasing the ...
At birth, people who carry a string of more than 40 CAG repeats within the first exon of the huntingtin gene are all but destined to develop Huntington’s disease. Yet, recent studies are converging on ...
Huntington's disease (HD) is an inherited neurodegenerative disease caused by an abnormal expansion of a CAG repeat in the huntingtin ( HTT) gene. The primary genetic determinant of the age at ...
People genetically susceptible to Huntington’s disease often see their movement ... This happens repeatedly until the CAG/CTG repeats expand enough to interfere with the function of the huntingtin ...
Huntington’s disease stems from a mutation in the HTT gene, which includes a repetitive sequence of DNA known as the CAG ...
This promising strategy could slow or halt Huntington's disease progression at the root of its genetic cause. "Targeting MSH3 is exciting not only because it's directly involved in the CAG repeat ...
How is it passed on? Huntington’s disease is a genetic disorder caused by an expansion of the CAG repeat region within the HTT gene. It exhibits an autosomal dominant pattern of inheritance ...
Latus will share progress toward the development of a novel, investigational, AAV drug candidate that is intended to address somatic instability that drives the CAG repeat expansion in HD.
Ranum, PhD Day: Wednesday, February 26, 2025 Abstract Title: Targeting MSH3 for Huntington’s Disease: Preclinical Validation of AAV-DB-3-miRNA to Prevent Somatic CAG Repeat Expansion Presenter ...