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Article

A quantitative systems pharmacology (QSP) platform for preclinical to clinical translation of in-vivo CRISPR-Cas therapy

20 Sept 2024

Abstract excerpt

Background: In-vivo CRISPR Cas genome editing is a complex therapy involving lipid nanoparticle (LNP), messenger RNA (mRNA), and single guide RNA (sgRNA). This novel modality requires prior modeling to predict dose-exposure-response relationships due to limited information on sgRNA and mRNA biodistribution. This work presents a QSP model to characterize, predict, and translate the...

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Share to Advanced biosensing and bioanalysis techniques
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 39372210
DOI: 10.3389/fphar.2024.1454785

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