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- W2913461168 abstract "Huntington’s disease (HD)is a devastating neurodegenerative disease caused by a single mutation, a CAGexpansion, in the huntingtin (HTT) gene. The resultant mutant HTT protein hasbeen shown to be the predominant toxic entity in the HD pathogenesis andtherapeutic strategies that aim to lower the mutant HTT show a great promise.The main objective of this work is to demonstrate a preclinical efficacy of anadeno-associated virus (AAV)-delivery of micro (mi)RNA-based gene therapy forthe treatment of HD. We have tested various therapeutic miRNAs to achieveoverall HTT protein lowering in HD rodent models and induced pluripotent stemcell (iPSC)-derived HD patient neuronal cultures. Excitingly, we havedemonstrated HTT lowering by the AAV5-miHTT in all HD models tested so far withno undesired events, which strongly supported the continuation of preclinicaltesting in large animals. Furthermore, we provided an evidence suggesting thattherapeutic miRNAs can be also active in the nucleus, extending their range ofapplicability. The possibility to use exosome-enriched vesicles as carriers ofpharmacokinetic/pharmacodynamic (PK/PD) measures for the AAV5-miHTT genetherapy, that would signal the presence of the active therapeutic miRNAs in thebrain, was further explored in preparation for a first clinical trial inhumans." @default.
- W2913461168 created "2019-02-21" @default.
- W2913461168 creator A5002010032 @default.
- W2913461168 date "2019-01-24" @default.
- W2913461168 modified "2023-09-23" @default.
- W2913461168 title "MicroRNA-based gene therapy for Huntington's disease : Silencing the villain" @default.
- W2913461168 hasPublicationYear "2019" @default.
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