Functional intercellular transmission of mihtt via extracellular vesicles: an in vitro proof-of-mechanism study

HIGHLIGHTS

  • who: Roberto D. V. S. Morais et al. from the Department of Research and Development, uniQure Biopharma BV, BP Amsterdam, The Netherlands Department of Gastroenterology and Hepatology, Leiden University Medical Center have published the research work: Functional Intercellular Transmission of miHTT via Extracellular Vesicles: An In Vitro Proof-of-Mechanism Study, in the Journal: Cells 2022, 11, 2748. of /2022/
  • what: The authors provide evidence that an engineered miRNA-miHTT-is loaded into EVs transported across extracellular space and taken up by neighboring cells and importantly that miHTT is active in recipient cells downregulating HTT . . .

     

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