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Regulatory-Compliant Validation of a Highly Sensitive qPCR for Biodistribution Assessment of Hemophilia A Patient Cells.


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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2023-12-31 | GSE237057 | GEO