AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes
Huntington disease (HD) is a fatal neurodegenerative genetic disorder, thought to reflect a toxic gain of function in huntingtin (Htt) protein. Adeno-associated viral vector serotype 5 (AAV5)- microRNA targeting huntingtin (miHTT) is a HD gene-therapy candidate that efficiently lowers HTT using RNAi...
Bewaard in:
| Hoofdauteurs: | , , , , , , , , |
|---|---|
| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
Elsevier
2019-12-01
|
| Reeks: | Molecular Therapy: Methods & Clinical Development |
| Online toegang: | http://www.sciencedirect.com/science/article/pii/S2329050119301081 |
| Tags: |
Geen labels, Wees de eerste die dit record labelt!
|