Unexpected CRISPR off-target mutation pattern in vivo are not typically germline-like [preprint]
UMass Chan AffiliationsDepartment of Biochemistry and Molecular Pharmacology
CRISPR-based gene therapy
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AbstractA computationally evolutionary investigation was performed to re-analyze the WGS data of the two studies published in Nature Methods (2015, 2017) with opposite conclusions on CRISPR off-target mutations. Our analysis concluded that the so-called unexpected SNVs pattern obtained by the study of Schaefer et al. are not typically germline-like. Some of unusual and unidentified mutations may arise, but the real reasons remain to be explored. Based on the available data and a direct comparison of the two studies, we presented two possible reasons and future re-analysis directions that may contribute to such different conclusions. To characterize the authentic CRISPR-mediated mutations, we are required to have appropriate controls to rule out other sources of mutations, which will be needed for benchmarking of targeting safety of CRISPR-based gene therapy.
bioRxiv 193565; doi: https://doi.org/10.1101/193565. Link to preprint on bioRxiv service.
Permanent Link to this Itemhttp://hdl.handle.net/20.500.14038/29264
RightsThe copyright holder for this preprint (which was not peer-reviewed) is the author/funder. It is made available under a CC-BY-NC-ND 4.0 International license.
Except where otherwise noted, this item's license is described as The copyright holder for this preprint (which was not peer-reviewed) is the author/funder. It is made available under a CC-BY-NC-ND 4.0 International license.