Secreted therapeutics: monitoring durability of microRNA-based gene therapies in the central nervous system

The preclinical development of microRNA-based gene therapies for inherited neurodegenerative diseases is accompanied by translational challenges. Due to the inaccessibility of the brain to periodically evaluate therapy effects, accessible and reliable biomarkers indicative of dosing, durability and...

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Bibliografiska uppgifter
I publikationen:Brain Commun
Huvudupphovsmän: Sogorb-Gonzalez, Marina, Vendrell-Tornero, Carlos, Snapper, Jolanda, Stam, Anouk, Keskin, Sonay, Miniarikova, Jana, Spronck, Elisabeth A, de Haan, Martin, Nieuwland, Rienk, Konstantinova, Pavlina, van Deventer1, Sander J, Evers, Melvin M, Vallès, Astrid
Materialtyp: Artigo
Språk:Inglês
Publicerad: Oxford University Press 2021
Ämnen:
Original Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC8093922/
https://ncbi.nlm.nih.gov/pubmed/34704020
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/braincomms/fcab054
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