December 3, 2021 Journal article Open Access

Pallarès-Masmitjà, Maria; Ivančić, Dimitrije; Mir-Pedrol, Júlia; Jaraba-Wallace, Jessica; Tagliani, Tommaso; Oliva, Baldomero; Rahmeh, Amal; Sánchez-Mejías, Avencia; Güell, Marc

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  "doi": "10.1038/s41467-021-27183-x", 
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    "doi": "10.1038/s41467-021-27183-x", 
    "description": "<p><strong>Abstract</strong></p>\n\n<p>While multiple technologies for small allele genome editing exist, robust technologies for targeted integration of large DNA fragments in mammalian genomes are still missing. Here we develop a gene delivery tool (FiCAT) combining the precision of a CRISPR-Cas9 (find module), and the payload transfer efficiency of an engineered piggyBac transposase (cut-and-transfer module). FiCAT combines the functionality of Cas9 DNA scanning and targeting DNA, with piggyBac donor DNA processing and transfer capacity. PiggyBac functional domains are engineered providing increased on-target integration while reducing off-target events. We demonstrate efficient delivery and programmable insertion of small and large payloads in cellulo (human (Hek293T, K-562) and mouse (C2C12)) and in vivo in mouse liver. Finally, we evolve more efficient versions of FiCAT by generating a targeted diversity of 394,000 variants and undergoing 4 rounds of evolution. In this work, we develop a precise and efficient targeted insertion of multi kilobase DNA fragments in mammalian genomes.</p>", 
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      "id": "CC-BY-4.0"
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    "title": "Find and cut-and-transfer (FiCAT) mammalian genome engineering", 
    "journal": {
      "volume": "12", 
      "title": "Nature Communications"
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        "title": "Unlocking Precision Gene Therapy", 
        "acronym": "UPGRADE", 
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    "publication_date": "2021-12-03", 
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        "orcid": "0000-0002-7317-7793", 
        "affiliation": "Department of Health and Experimental Sciences, Pompeu Fabra University, Barcelona, Spain", 
        "name": "Pallar\u00e8s-Masmitj\u00e0,\u00a0Maria"
      }, 
      {
        "affiliation": "Department of Health and Experimental Sciences, Pompeu Fabra University, Barcelona, Spain - Barcelona Institute of Science and Technology, Barcelona, Spain", 
        "name": "Ivan\u010di\u0107,\u00a0Dimitrije"
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      {
        "orcid": "0000-0001-6104-9260", 
        "affiliation": "Department of Health and Experimental Sciences, Pompeu Fabra University, Barcelona, Spain", 
        "name": "Mir-Pedrol,\u00a0J\u00falia"
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      {
        "affiliation": "Department of Health and Experimental Sciences, Pompeu Fabra University, Barcelona, Spain", 
        "name": "Jaraba-Wallace,\u00a0Jessica"
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      {
        "affiliation": "Department of Health and Experimental Sciences, Pompeu Fabra University, Barcelona, Spain", 
        "name": "Tagliani,\u00a0Tommaso"
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        "orcid": "0000-0003-0702-0250", 
        "affiliation": "Department of Health and Experimental Sciences, Pompeu Fabra University, Barcelona, Spain", 
        "name": "Oliva,\u00a0Baldomero"
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        "affiliation": "Department of Health and Experimental Sciences, Pompeu Fabra University, Barcelona, Spain", 
        "name": "Rahmeh,\u00a0Amal"
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        "affiliation": "Department of Health and Experimental Sciences, Pompeu Fabra University, Barcelona, Spain", 
        "name": "S\u00e1nchez-Mej\u00edas,\u00a0Avencia"
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        "affiliation": "Department of Health and Experimental Sciences, Pompeu Fabra University, Barcelona, Spain", 
        "name": "G\u00fcell,\u00a0Marc"
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