<?xml version="1.0" encoding="UTF-8"?>
<STUDY_SET xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance">
  <STUDY center_name="BioProject" alias="PRJNA834963" accession="SRP373554">
    <IDENTIFIERS>
      <PRIMARY_ID>SRP373554</PRIMARY_ID>
      <EXTERNAL_ID namespace="BioProject" label="primary">PRJNA834963</EXTERNAL_ID>
    </IDENTIFIERS>
    <DESCRIPTOR>
      <STUDY_TITLE>Off-target cleavage analysis of AAVS1-specific Cas9 nucleases</STUDY_TITLE>
      <STUDY_TYPE existing_study_type="Other"/>
      <STUDY_ABSTRACT>In this study, we show that fully viral gene-deleted adenoviral vector delivery of donors prone to HMEJ and HR, together with matched CRISPR-Cas9 complexes, achieves targeted chromosomal integration of large genetic payloads in human cells. This was exploit for the genetic complementation of defective DMD genes in human muscle progenitor cells.</STUDY_ABSTRACT>
    </DESCRIPTOR>
  </STUDY>
</STUDY_SET>
