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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  <identifier identifierType="URL">https://zenodo.org/record/6104372</identifier>
  <creators>
    <creator>
      <creatorName>Pallarès-Masmitjà, Maria</creatorName>
      <givenName>Maria</givenName>
      <familyName>Pallarès-Masmitjà</familyName>
      <nameIdentifier nameIdentifierScheme="ORCID" schemeURI="http://orcid.org/">0000-0002-7317-7793</nameIdentifier>
      <affiliation>Department of Health and Experimental Sciences, Pompeu Fabra University, Barcelona, Spain</affiliation>
    </creator>
    <creator>
      <creatorName>Ivančić, Dimitrije</creatorName>
      <givenName>Dimitrije</givenName>
      <familyName>Ivančić</familyName>
      <affiliation>Department of Health and Experimental Sciences, Pompeu Fabra University, Barcelona, Spain - Barcelona Institute of Science and Technology, Barcelona, Spain</affiliation>
    </creator>
    <creator>
      <creatorName>Mir-Pedrol, Júlia</creatorName>
      <givenName>Júlia</givenName>
      <familyName>Mir-Pedrol</familyName>
      <nameIdentifier nameIdentifierScheme="ORCID" schemeURI="http://orcid.org/">0000-0001-6104-9260</nameIdentifier>
      <affiliation>Department of Health and Experimental Sciences, Pompeu Fabra University, Barcelona, Spain</affiliation>
    </creator>
    <creator>
      <creatorName>Jaraba-Wallace, Jessica</creatorName>
      <givenName>Jessica</givenName>
      <familyName>Jaraba-Wallace</familyName>
      <affiliation>Department of Health and Experimental Sciences, Pompeu Fabra University, Barcelona, Spain</affiliation>
    </creator>
    <creator>
      <creatorName>Tagliani, Tommaso</creatorName>
      <givenName>Tommaso</givenName>
      <familyName>Tagliani</familyName>
      <affiliation>Department of Health and Experimental Sciences, Pompeu Fabra University, Barcelona, Spain</affiliation>
    </creator>
    <creator>
      <creatorName>Oliva, Baldomero</creatorName>
      <givenName>Baldomero</givenName>
      <familyName>Oliva</familyName>
      <nameIdentifier nameIdentifierScheme="ORCID" schemeURI="http://orcid.org/">0000-0003-0702-0250</nameIdentifier>
      <affiliation>Department of Health and Experimental Sciences, Pompeu Fabra University, Barcelona, Spain</affiliation>
    </creator>
    <creator>
      <creatorName>Rahmeh, Amal</creatorName>
      <givenName>Amal</givenName>
      <familyName>Rahmeh</familyName>
      <affiliation>Department of Health and Experimental Sciences, Pompeu Fabra University, Barcelona, Spain</affiliation>
    </creator>
    <creator>
      <creatorName>Sánchez-Mejías, Avencia</creatorName>
      <givenName>Avencia</givenName>
      <familyName>Sánchez-Mejías</familyName>
      <affiliation>Department of Health and Experimental Sciences, Pompeu Fabra University, Barcelona, Spain</affiliation>
    </creator>
    <creator>
      <creatorName>Güell, Marc</creatorName>
      <givenName>Marc</givenName>
      <familyName>Güell</familyName>
      <nameIdentifier nameIdentifierScheme="ORCID" schemeURI="http://orcid.org/">0000-0003-4000-7912</nameIdentifier>
      <affiliation>Department of Health and Experimental Sciences, Pompeu Fabra University, Barcelona, Spain</affiliation>
    </creator>
  </creators>
  <titles>
    <title>Find and cut-and-transfer (FiCAT) mammalian genome engineering</title>
  </titles>
  <publisher>Zenodo</publisher>
  <publicationYear>2021</publicationYear>
  <dates>
    <date dateType="Issued">2021-12-03</date>
  </dates>
  <resourceType resourceTypeGeneral="JournalArticle"/>
  <alternateIdentifiers>
    <alternateIdentifier alternateIdentifierType="url">https://zenodo.org/record/6104372</alternateIdentifier>
  </alternateIdentifiers>
  <relatedIdentifiers>
    <relatedIdentifier relatedIdentifierType="DOI" relationType="IsIdenticalTo">10.1038/s41467-021-27183-x</relatedIdentifier>
    <relatedIdentifier relatedIdentifierType="URL" relationType="IsPartOf">https://zenodo.org/communities/upgrade-h2020-project</relatedIdentifier>
  </relatedIdentifiers>
  <rightsList>
    <rights rightsURI="https://creativecommons.org/licenses/by/4.0/legalcode">Creative Commons Attribution 4.0 International</rights>
    <rights rightsURI="info:eu-repo/semantics/openAccess">Open Access</rights>
  </rightsList>
  <descriptions>
    <description descriptionType="Abstract">&lt;p&gt;&lt;strong&gt;Abstract&lt;/strong&gt;&lt;/p&gt;

&lt;p&gt;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.&lt;/p&gt;</description>
  </descriptions>
  <fundingReferences>
    <fundingReference>
      <funderName>European Commission</funderName>
      <funderIdentifier funderIdentifierType="Crossref Funder ID">10.13039/100010661</funderIdentifier>
      <awardNumber awardURI="info:eu-repo/grantAgreement/EC/H2020/825825/">825825</awardNumber>
      <awardTitle>Unlocking Precision Gene Therapy</awardTitle>
    </fundingReference>
  </fundingReferences>
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