Researchers have reported the first evidence that gene therapy can slow the progression of Huntington’s disease in humans, according to Nature. From a genetics standpoint, Huntington’s disease (HD) is caused by a repeat expansion in the HTT gene, and it has long been considered a high-barrier therapeutic target because the brain is difficult to access and neural degeneration is progressive over time. The fact that a gene therapy trial is now showing the clearest evidence yet of slowing the disease marks a significant step forward.
This development raises several key questions. For instance, what vector and delivery method were used to transport the therapy into the central nervous system (CNS)? Additionally, what genetic or epigenetic barriers still need to be addressed for broader success? If this approach ultimately proves effective, it could expand the potential for treating other neuro-genetic conditions such as certain forms of ALS or spinocerebellar ataxias. For our class, this story highlights how the combination of genotype (a repeat expansion) and phenotype (neurodegeneration) can be modified at the molecular level. It also emphasizes how central genetics is to the development of emerging therapies.
In sum, this advancement represents a major milestone: a shift from gene therapy being used primarily in blood or immune disorders to meaningful progress in treating serious neurological diseases. This a very relevant piece of genetics news.
Resources
Dolgin, Elie. “Huntington’s Disease Treated for First Time Using Gene Therapy.” Nature, 25 Sept. 2025, https://www.nature.com/articles/d41586-025-03139-9
Piao, Xuejiao, et al. “Advances in Gene and Cellular Therapeutic Approaches for Huntington’s Disease.” Protein & Cell, 2025, https://pubmed.ncbi.nlm.nih.gov/39121016
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