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Widespread and sustained target engagement in Huntington’s disease minipigs upon intrastriatal microRNA-based gene therapy

Authors: Astrid Vallès; Melvin M. Evers; Anouk Stam; Marina Sogorb-Gonzalez; Cynthia Brouwers; Carlos Vendrell-Tornero; Seyda Acar-Broekmans; +16 Authors

Widespread and sustained target engagement in Huntington’s disease minipigs upon intrastriatal microRNA-based gene therapy

Abstract

Striatal delivery of microRNA-gene therapy results in widespread brain huntingtin protein lowering in Huntington’s disease minipigs up to 1 year.

Keywords

Huntingtin Protein, Swine, Genetic Vectors, Genetic Therapy, Disease Models, Animal, MicroRNAs, Huntington Disease, Animals, Humans, Swine, Miniature, Tissue Distribution

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    popularity
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    Top 1%
    influence
    This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
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Powered by OpenAIRE graph
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selected citations
These citations are derived from selected sources.
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
45
Top 1%
Top 10%
Top 1%
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