Summary
The formulation of drug nanocarriers has attracted increasing attention over the last decades. In particular, the possibility to combine
therapeutic and imaging functionalities in a single nanoplatform (theranostic) has been widely explored to advance therapeutic
approaches and promote the transition from conventional medicine to personalized medicine.
Drug nanocarriers, holding the potential to produce a revolution in medicine, are lipid nanoparticles (LNPs) for gene delivery. They
have proven versatility and efficacy even though today it is still not possible to predict and control their cellular uptake and targeted
delivery. One of the main unmet challenges of LNP formulations is increasing their cellular uptake and gene release into the cytosol.
Currently, it is possible to follow the fate of LNPs after administration only with the addition of labels (e.g., fluorophore) or specific
ribonucleic acid (RNA) sequence encoding for fluorescent protein. This results in the lack of possibility to follow the fate of LNPs once
administered in vivo with a non-invasive imaging technique. Theranostic strategies to deliver RNA and simultaneous imaging of
target organs is an important unmet goal.
Recently, it has been demonstrated that fluorination of components in gene delivery vehicles strongly improved their cellular uptake
and, most importantly, their gene endosomal escape. The fluorination strategies investigated so far use either -CF3 groups or long
linear perfluoroalkyl chains, but a tailored design of the fluorinated group could also endow the nanocarrier with excellent imaging
functions. In this context, FluoNeeD strategy is to tailor fluorination of LNPs in order to: (i) improve their therapeutic efficacy by
enhancing LNP cellular uptake and promoting gene endosomal escape; (ii) render LNPs trackable in vivo by 19F-MRI and in vitro/ex vivo by Raman microscopy.
therapeutic and imaging functionalities in a single nanoplatform (theranostic) has been widely explored to advance therapeutic
approaches and promote the transition from conventional medicine to personalized medicine.
Drug nanocarriers, holding the potential to produce a revolution in medicine, are lipid nanoparticles (LNPs) for gene delivery. They
have proven versatility and efficacy even though today it is still not possible to predict and control their cellular uptake and targeted
delivery. One of the main unmet challenges of LNP formulations is increasing their cellular uptake and gene release into the cytosol.
Currently, it is possible to follow the fate of LNPs after administration only with the addition of labels (e.g., fluorophore) or specific
ribonucleic acid (RNA) sequence encoding for fluorescent protein. This results in the lack of possibility to follow the fate of LNPs once
administered in vivo with a non-invasive imaging technique. Theranostic strategies to deliver RNA and simultaneous imaging of
target organs is an important unmet goal.
Recently, it has been demonstrated that fluorination of components in gene delivery vehicles strongly improved their cellular uptake
and, most importantly, their gene endosomal escape. The fluorination strategies investigated so far use either -CF3 groups or long
linear perfluoroalkyl chains, but a tailored design of the fluorinated group could also endow the nanocarrier with excellent imaging
functions. In this context, FluoNeeD strategy is to tailor fluorination of LNPs in order to: (i) improve their therapeutic efficacy by
enhancing LNP cellular uptake and promoting gene endosomal escape; (ii) render LNPs trackable in vivo by 19F-MRI and in vitro/ex vivo by Raman microscopy.
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More information & hyperlinks
| Web resources: | https://cordis.europa.eu/project/id/101108183 |
| Start date: | 01-06-2024 |
| End date: | 31-08-2026 |
| Total budget - Public funding: | - 212 163,00 Euro |
Cordis data
Original description
The formulation of drug nanocarriers has attracted increasing attention over the last decades. In particular, the possibility to combinetherapeutic and imaging functionalities in a single nanoplatform (theranostic) has been widely explored to advance therapeutic
approaches and promote the transition from conventional medicine to personalized medicine.
Drug nanocarriers, holding the potential to produce a revolution in medicine, are lipid nanoparticles (LNPs) for gene delivery. They
have proven versatility and efficacy even though today it is still not possible to predict and control their cellular uptake and targeted
delivery. One of the main unmet challenges of LNP formulations is increasing their cellular uptake and gene release into the cytosol.
Currently, it is possible to follow the fate of LNPs after administration only with the addition of labels (e.g., fluorophore) or specific
ribonucleic acid (RNA) sequence encoding for fluorescent protein. This results in the lack of possibility to follow the fate of LNPs once
administered in vivo with a non-invasive imaging technique. Theranostic strategies to deliver RNA and simultaneous imaging of
target organs is an important unmet goal.
Recently, it has been demonstrated that fluorination of components in gene delivery vehicles strongly improved their cellular uptake
and, most importantly, their gene endosomal escape. The fluorination strategies investigated so far use either -CF3 groups or long
linear perfluoroalkyl chains, but a tailored design of the fluorinated group could also endow the nanocarrier with excellent imaging
functions. In this context, FluoNeeD strategy is to tailor fluorination of LNPs in order to: (i) improve their therapeutic efficacy by
enhancing LNP cellular uptake and promoting gene endosomal escape; (ii) render LNPs trackable in vivo by 19F-MRI and in vitro/ex vivo by Raman microscopy.
Status
SIGNEDCall topic
HORIZON-MSCA-2022-PF-01-01Update Date
31-07-2023
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