Let’s step back and review the technologies now available to treat this neurodegenerative disease, caused by a gene producing a toxic protein. Antisense approaches, under development for decades and now in clinical trials, shut off the problematic gene. However, this type of treatment would need to be regularly delivered to nervous system tissues. Gene editing — not in the clinic yet — could actually remove the gene from somatic cells in affected individuals.
Emory researchers developed the pig HD model in collaboration with colleagues in Guangzhou, and anticipate it will be a practical way to test treatments such as gene editing. In comparison with mice, delivery to affected nervous system tissues can be better tested in pigs, because their size is closer to that of humans. The pig model of HD, published yesterday in Cell, also more closely matches the symptoms of the human disease. This research was covered by Chinese media organizations.
- MIT Technology Review round up of gene editing approaches
- Cure HD blog describing how Roche and Ionis are gearing up for a pivotal phase III clinical trial of their antisense drug. See coverage by Endpoints News,
- Jump from the sauna into the snow by reminding yourself that “breakthrough” is a premature hype word (Health News Review).
- Longer article from Mosaic