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Showing 1–12 of 12 results
Advanced filters: Author: Samira Kiani Clear advanced filters

  • 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
    P: 1-20

  • 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

  • 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

  • Transcription activator–like effector nucleases (TALENs) are a new technology for modifying the genome at specific loci of interest. Hockemeyer et al. now demonstrate the utility of TALENs for gene targeting in human pluripotent stem cells.

    • Dirk Hockemeyer
    • Haoyi Wang
    • Rudolf Jaenisch
    Research
    Nature Biotechnology
    Volume: 29, P: 731-734

  • 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

  • This Perspective discusses how the Somatic Cell Genome Editing Consortium aims to accelerate the implementation of safe and effective genome-editing therapies in the clinic.

    • Krishanu Saha
    • Erik J. Sontheimer
    • Jiangbing Zhou
    ReviewsOpen Access
    Nature
    Volume: 592, P: 195-204

  • There has been limited success in generating tissues from human induced pluripotent stem cells (hiPSCs). Here, the authors genetically engineer expression of the transcription factor Gata6 in a single isogenic hiPSC population resulting in complex tissue structures that exhibit liver bud-like properties.

    • Patrick Guye
    • Mohammad R. Ebrahimkhani
    • Ron Weiss
    ResearchOpen Access
    Nature Communications
    Volume: 7, P: 1-12

  • The length of a single guide RNA (gRNA) determines the function of Cas9. In this study 20-nt gRNAs allowed nuclease activity and genome editing, whereas 14-nt gRNAs mediated transcriptional activation or repression.

    • Samira Kiani
    • Alejandro Chavez
    • George Church
    Research
    Nature Methods
    Volume: 12, P: 1051-1054

  • 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

  • The fusion of three transcriptional activation domains to a nuclease-deficient Cas9 achieves robust induction of gene expression and can induce differentiation of hiPSCs.

    • Alejandro Chavez
    • Jonathan Scheiman
    • George M Church
    Research
    Nature Methods
    Volume: 12, P: 326-328