Article ID Journal Published Year Pages File Type
10885933 Drug Discovery Today 2014 5 Pages PDF
Abstract

- 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.
Related Topics
Life Sciences Biochemistry, Genetics and Molecular Biology Biotechnology
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