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  1. 8 de dez. de 2020 · We proposed an effective method to introduce Cas9 into the AAVS1 locus using viral vectors. Stably expressed Cas9, especially Cas9 nickase, in the safe harbor can be used for precise gene...

    • Hideki Hayashi, Yoshinao Kubo, Mai Izumida, Toshifumi Matsuyama
    • 2020
  2. 24 de jan. de 2022 · We describe a pipeline to computationally predict and experimentally validate human genomic safe harbor sites and characterize two sites, Rogi1 and Rogi2, suitable for integrative gene delivery.

  3. 20 de fev. de 2024 · Open access. Published: 20 February 2024. Harnessing eukaryotic retroelement proteins for transgene insertion into human safe-harbor loci. Xiaozhu Zhang, Briana Van Treeck, Connor A. Horton,...

  4. 29 de mar. de 2023 · We first screened a panel of guide sequences targeting the AAVS1 safe harbor locus via a plasmid-to-plasmid integration assay, in which we cloned 32-bp target sites derived from AAVS1 into pTarget ...

  5. 1 de jul. de 2019 · Abstract. This study identified 35 new sites for targeted transgene insertion that have the potential to serve as new human genomic “safe harbor” sites (SHS). SHS potential for these 35 sites, located on 16 chromosomes, including both arms of the human X chromosome, and for the existing human SHS AAVS1, hROSA26, and CCR5 was ...

  6. In this study, we have developed a non–viral vector–based engineering system for large DNA multimodality reporter gene integration into the AAVS1 safe harbor genomic locus. To improve safety further, we used MCs as the DNA vector of choice, which eliminates bacterial DNA contamination and antibiotic resistance genes.

    • John J. Kelly, Moe Saee-Marand, Nivin N. Nyström, Melissa M. Evans, Yuanxin Chen, Francisco M. Marti...
    • 2021
  7. 15 de mai. de 2016 · As disruption of PPP1R12C is not associated with any known disease, the AAVS1 locus is often considered a safe-harbor for transgene targeting. Rivaling transgenic approaches based on simple viral transduction or transposition, targeted transgene integration with nucleases must be efficient and standardized.