Impact of Chemical Composition on the Nanostructure and Biological Activity of α-Galactosidase-Loaded Nanovesicles for Fabry Disease Treatment

Tomsen-Melero, Judit; Passemard, Solène; García‐Aranda, Natalia; Díaz‐Riascos, Zamira V.; González-Rioja, Ramon; Pedersen, Jannik Nedergaard; Lyngsø, Jeppe; Merlo-Mas, Josep; Cristóbal-Lecina, Edgar; Corchero, José Luís; Pulido, Daniel; Cámara-Sánchez, Patricia; Portnaya, Irina; Ionita, Inbal; Veciana, Jaume; Sala, Santiago; Royo, Míriam; Córdoba, Alba; Danino, Dganit; Pedersen, Jan Skov; González‐Mira, Elisabet; Abasolo, Ibane; Ventosa, Nora

doi:10.1021/acsami.0c16871

Cited by 25 publications

(13 citation statements)

References 55 publications

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“…It seems that this MKC amount is not enough to stabilize such highly concentrated samples. However, higher MKC concentrations, although could improve the stability of these concentrated samples, it also could entail a toxicological concern as well as the abolishment of the enzymatic activity of the GLA, as reported by Tomsen-Melero et al [7] .…”

Section: Resultsmentioning

confidence: 87%

“…An experimental design was constructed to study the influence of the formulation (independent variables or factors) on the physicochemical properties (dependent variables or responses) of intermediate nanoGLA obtained by DELOS-susp, as shown in Table 3 . Low and high levels for each factor ( Table 2 ) were selected based on previous experiments and studies [4] , [7] .…”

Section: Resultsmentioning

confidence: 99%

“…Moreover, the addition of a targeting peptide on the liposomal membrane can facilitate cellular up-take via peptide receptors recognition and could help to improve GLA biodistribution. Thus, GLA-loaded nanoliposomes (nanoGLA) functionalized with Arginine-Glycine-Aspartic acid (RGD) peptide and prepared by DELOS-susp, have recently been reported to increase enzymatic activity and intracellular penetration, showing the great potential of this CO 2 -based methodology for the simple production of protein-nanoliposome therapeutic conjugates [4] , [7] . Fig.…”

Section: Introductionmentioning

confidence: 99%

“…Besides, the enzyme entrapment efficiency in the liposomes was below 40% [4] . The addition of cationic surfactant myristalkonium chloride (MKC) into the formulation in low concentration allowed to increase the drug entrapment efficiency as well as the colloidal stability of the system [7] . However, further work was required in order to obtain a stable formulation with the proper protein concentration.…”

Section: Introductionmentioning

confidence: 99%

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Application of Quality by Design to the robust preparation of a liposomal GLA formulation by DELOS-susp method

Merlo-Mas¹,

Tomsen-Melero

Corchero

et al. 2021

The Journal of Supercritical Fluids

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Section: Resultsmentioning

confidence: 87%

Section: Resultsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

Application of Quality by Design to the robust preparation of a liposomal GLA formulation by DELOS-susp method

Merlo-Mas¹,

Tomsen-Melero

Corchero

et al. 2021

The Journal of Supercritical Fluids

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“…Nanocarriers improve bioavailability and ensure targeted drug transport, selective targeting of cancer cells, and controlled drug release [186]. To improve the stability of the liposome systems and to protect against capture by the immune system cells, liposomes were modified by the ionic or non-ionic surfactants and/or polymers incorporation into phospholipid membrane [187].…”

Section: Liposomal Systems Based On Amphiphilic Triflates Of Acyloxypropylphosphonium and L-α-phosphatidylcholinementioning

confidence: 99%

Rational Design 2-Hydroxypropylphosphonium Salts as Cancer Cell Mitochondria-Targeted Vectors: Synthesis, Structure, and Biological Properties

et al. 2021

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It has been shown for a wide range of epoxy compounds that their interaction with triphenylphosphonium triflate occurs with a high chemoselectivity and leads to the formation of (2-hydroxypropyl)triphenylphosphonium triflates 3 substituted in the 3-position with an alkoxy, alkylcarboxyl group, or halogen, which were isolated in a high yield. Using the methodology for the disclosure of epichlorohydrin with alcohols in the presence of boron trifluoride etherate, followed by the substitution of iodine for chlorine and treatment with triphenylphosphine, 2-hydroxypropyltriphenylphosphonium iodides 4 were also obtained. The molecular and supramolecular structure of the obtained phosphonium salts was established, and their high antitumor activity was revealed in relation to duodenal adenocarcinoma. The formation of liposomal systems based on phosphonium salt 3 and L-α-phosphatidylcholine (PC) was employed for improving the bioavailability and reducing the toxicity. They were produced by the thin film rehydration method and exhibited cytotoxic properties. This rational design of phosphonium salts 3 and 4 has promising potential of new vectors for targeted delivery into mitochondria of tumor cells.

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Poly(Sitosterol)‐Based Hydrophobic Blocks in Amphiphilic Block Copolymers for the Assembly of Hybrid Vesicles

Brodszkij,

Ryberg,

Lyons

et al. 2024

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Amphiphilic block copolymer and lipids can be assembled into hybrid vesicles (HVs), which are an alternative to liposomes and polymersomes. Block copolymers that have either poly(sitostryl methacrylate) or statistical copolymers of sitosteryl methacrylate and butyl methacrylate as the hydrophobic part and a poly(carboxyethyl acrylate) hydrophilic segment are synthesized and characterized. These block copolymers assemble into small HVs with soybean L‐α‐phosphatidylcholine (soyPC), confirmed by electron microscopy and small‐angle X‐ray scattering. The membrane's hybrid nature is illustrated by fluorescence resonance energy transfer between labeled building blocks. The membrane packing, derived from spectra when using Laurdan as an environmentally sensitive fluorescent probe, is comparable between small HVs and the corresponding liposomes with molecular sitosterol, although the former show indications of transmembrane asymmetry. Giant HVs with homogenous distribution of the block copolymers and soyPC in their membranes are assembled using the electroformation method. The lateral diffusion of both building blocks is slowed down in giant HVs with higher block copolymer content, but their permeability toward (6)‐carboxy‐X‐rhodamine is higher compared to giant vesicles made of soyPC and molecular sitosterol. This fundamental effort contributes to the rapidly expanding understanding of the integration of natural membrane constituents with designed synthetic compounds to form hybrid membranes.

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Impact of Chemical Composition on the Nanostructure and Biological Activity of α-Galactosidase-Loaded Nanovesicles for Fabry Disease Treatment

Cited by 25 publications

References 55 publications

Application of Quality by Design to the robust preparation of a liposomal GLA formulation by DELOS-susp method

Application of Quality by Design to the robust preparation of a liposomal GLA formulation by DELOS-susp method

Rational Design 2-Hydroxypropylphosphonium Salts as Cancer Cell Mitochondria-Targeted Vectors: Synthesis, Structure, and Biological Properties

Poly(Sitosterol)‐Based Hydrophobic Blocks in Amphiphilic Block Copolymers for the Assembly of Hybrid Vesicles

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