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.

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

Merlin S.
Methodology
;
Olgasi C.
Methodology
;
Follenzi A.
Penultimo
Conceptualization
;
2020-01-01

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.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11579/125433
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