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Showing 1–9 of 9 results
Advanced filters: Author: Mo R. Ebrahimkhani Clear advanced filters
  • Gain-of-function mutations in CTNNB1 (encoding for b-catenin) leading to deregulated Wnt/β-catenin signaling are frequently observed in patients with hepatocellular carcinoma (HCC). Here the authors show that inhibiting b-catenin with lipid nanoparticles encapsulating siRNA targeting CTNNB1 impairs tumor growth and promotes anti-tumor immunity in preclinical HCC models.

    • Brandon M. Lehrich
    • Evan R. Delgado
    • Satdarshan P. Monga
    ResearchOpen Access
    Nature Communications
    Volume: 16, P: 1-26
  • Possible immunogenicity of the Cas9 protein raises concerns about therapeutic applications. Here the authors identify pre-existing CD8+T-cell immunity in healthy individuals and in response modify Cas9 to remove the immunodominant epitopes.

    • Shayesteh R. Ferdosi
    • Radwa Ewaisha
    • Karen S. Anderson
    ResearchOpen Access
    Nature Communications
    Volume: 10, P: 1-10
  • Moghadam et al. developed a CRISPR transcriptional repressor to silence MyD88 expression in vivo to modulate immune response against AAV gene therapy and septicaemia.

    • Farzaneh Moghadam
    • Ryan LeGraw
    • Samira Kiani
    Research
    Nature Cell Biology
    Volume: 22, P: 1143-1154
  • A genetically inducible stem cell-derived embryoid model of early post-implantation human embryogenesis captures the codevelopment of embryonic tissue and extra-embryonic endoderm and mesoderm niche with early haematopoiesis, with potential for drug testing and disease modelling.

    • Joshua Hislop
    • Qi Song
    • Mo R. Ebrahimkhani
    ResearchOpen Access
    Nature
    Volume: 626, P: 367-376
  • CRISPR-based genome editing therapeutics are entering the clinic, but in vitro and in vivo tools are needed to assess their safety and efficacy. The authors review complementary technologies to monitor the biological effects of genome editing across scales, including the direct measurement of editing outcomes in DNA, human microphysiological systems and non-invasive in vivo imaging.

    • Benjamin S. Freedman
    • Jeff W. M. Bulte
    • Shengdar Q. Tsai
    Reviews
    Nature Reviews Genetics
    Volume: 27, P: 323-342
  • The fusion of dead Cas9 with KRAB and the transcriptional repressor domain of the chromatin modifier MeCP2 leads to an efficient transcriptional silencer that can be applied to genome-scale screens and genetic circuits.

    • Nan Cher Yeo
    • Alejandro Chavez
    • George M. Church
    Research
    Nature Methods
    Volume: 15, P: 611-616
  • Human microphysiology systems (MPS) have evolved as experimental model systems. This Review explores these so-called organ-on-a-chip systems and the role of biomimetic human liver MPS in drug development and precision medicine, providing insights into their design and use as models of liver physiology and disease.

    • Albert Gough
    • Alejandro Soto-Gutierrez
    • D. Lansing Taylor
    Reviews
    Nature Reviews Gastroenterology & Hepatology
    Volume: 18, P: 252-268