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Showing 1–5 of 5 results
Advanced filters: Author: Matthew Gemberling Clear advanced filters
  • An injury-dependent enhancer element is identified that activates gene expression in regenerating zebrafish tissues and can be engineered into DNA constructs that increase tissue regenerative capacity; the element is also active in injured mouse tissue.

    • Junsu Kang
    • Jianxin Hu
    • Kenneth D. Poss
    Research
    Nature
    Volume: 532, P: 201-206
  • Repression of gene transcription using CRISPR-Cas9 has been achieved in vitro but not for delivery into adult animal models. Here, the authors use AAV8 to deliver the transcriptional repressor dSaCas9KRAB to the cholesterol regulator Pcsk9, and show repression up to 24 weeks and reduced cholesterol levels in mice.

    • Pratiksha I. Thakore
    • Jennifer B. Kwon
    • Charles A. Gersbach
    ResearchOpen Access
    Nature Communications
    Volume: 9, P: 1-9
  • In the mdx mouse model of Duchenne muscular dystrophy, single intravenous administration of AAV-CRISPR–Cas9 vectors provides efficient genome editing and restoration of dystrophin expression lasting for one year.

    • Christopher E. Nelson
    • Yaoying Wu
    • Charles A. Gersbach
    Research
    Nature Medicine
    Volume: 25, P: 427-432
  • Loss-of-function (LOF) approaches are powerful experimental tools for characterizing gene functions. However, emerging discrepancies when genes are investigated using different tools or organisms has triggered debate about how such LOF results should be biologically interpreted. In this Review, experts from varied fields discuss how understanding the underlying features of each LOF approach can provide explanations for different experimental outcomes and can guide their optimal and reliable application.

    • Benjamin E. Housden
    • Matthias Muhar
    • Norbert Perrimon
    Reviews
    Nature Reviews Genetics
    Volume: 18, P: 24-40