Providing quantitative measurement for the intracellular delivery of therapeutic macromolecules.
Quantitative intracellular delivery, which holds promise for therapeutic control over major killers including cardiovascular diseases, genetic disorders and cancers, is hampered by inefficient cellular targeting and uptake of pharmaceutical drugs as a function of structural inconsistency of drug delivery vectors.
The need for a harmonised legislation and suitable standards for gene therapy products emphasises the impact this has on drug development and manufacturability. To a large extent, these issues are attributed to the lack of a measurement capability that can address factors influencing uncertainty and reproducibility of intracellular delivery, thereby enabling the systemic assessment of the safety and efficacy of drug and gene delivery technologies.
NPL is developing an advanced measurement capability for the quantitative characterisation of delivery vectors, their uptake to target cells and potential specificity of cell and tissue targeting. The capability is a reference methodology encompassing spectroscopy, spectrometry and microscopy methods to enable the validation of delivery measurands necessary for the formulation of future reference materials.
GeT peptides: a single-domain approach to gene delivery
Lamarre B, Ravi J, Ryadnov MG
Chem. Comm., 2011, 47, 9045-9047.
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