Cell-based therapies for Huntington's disease

- Specifically silencing mHTT can be accomplished by antisense oligonucleotides.
- Successes in stem cells and NPC studies move cell-based therapy forward.
- GET-NPT takes advantage of gene therapy in patient-derived cells for cell therapy.
- Noninvasively monitoring the transplanted cells in vivo is crucial.
- A transgenic HD monkey model is a suitable candidate for preclinical studies.