Beilstein J. Nanotechnol.2025,16, 435–463, doi:10.3762/bjnano.16.34
oligomer (PMO); phosphorothioate (PS); polyplexes; ribose substitutions; small interfering RNA (siRNA); synthetic polymers; tricyclo-DNA (tcDNA); Introduction
The development and use of personalised therapies tailored to individual patients have emerged as a powerful strategy for treating various
of cysteine-PNAs via disulfide linkage [126]. These compounds showed a PNA dose-dependent cell toxicity and exhibited up to almost 20-fold increased luciferase activity compared to free arginine-PNAs and dithiothreitol control. In another study, Kuhn et al. developed aminoethylene-based PMO
PMO conjugate containing linolenic acid with three double bonds (LenA) exhibited enhanced cellular internalisation while maintaining high cell viability on various cell lines, leading to the highest splice switching activity (Figure 8).
Poly(propylene imine). Since pioneering research by Vögtle and
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Figure 1:
Common chemical modifications used in antisense oligonucleotides (ASOs). First-generation ASOs invo...
Beilstein J. Nanotechnol.2012,3, 428–437, doi:10.3762/bjnano.3.49
in the benzene-PMO, due to the fact that the benzene-PMO walls possess a neutron scattering length density (SLD) similar to that of nitrogen in the condensed state. However, signals at higher q-values (>1 1/Å) are not affected with respect to their SANS intensity, even after complete pore filling
, confirming the assumption of a crystal-like periodicity within the PMO material walls due to π–π interactions between the organic bridges. The SLD of pristine benzene-PMO was altered by functionalizing the surface with different amounts of SO3H-groups, using the grafting method. For a low degree of
diffraction signals is no longer observed proving that homogeneously distributed SO3H-groups on the inner pore walls of the benzene-PMO alter the SLD in a way that it no longer fits to the SLD of the condensed N2.
Keywords: contrast matching; crystal-like periodicity; distribution of functional groups; PMO
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Scheme 1:
Illustration of the hexagonal pore arrangement of a functionalized benzene-PMO with the benzene rin...