Regulatory-Compliant Validation of a Highly Sensitive qPCR for Biodistribution Assessment of Hemophilia A Patient Cells.
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ABSTRACT: Bittorf and colleagues present the validation of a qPCR-based method to detect human genomic DNA in mouse organs following European and international guidelines. They further introduce a biodistribution study of a medicinal product consisting of lentiviral-transduced BOECs from hemophilic patients to investigate the toxicokinetic safety profile for clinical translation.
SUBMITTER: Bittorf P
PROVIDER: S-EPMC7327859 | biostudies-literature | 2020 Sep
REPOSITORIES: biostudies-literature
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