Optimization of Cationic Lipid Mediated Gene Transfer: Structure-Function, Physico-Chemical, and Cellular Studies

Carrière, Marie; Tranchant, Isabelle; Niore, Pierre-Antoine; Byk, Gérardo; Mignet, Nathalie; Escriou, Virginie; Scherman, Daniel; Herscovici, Jean

doi:10.1081/lpr-120004781

Cited by 14 publications

(9 citation statements)

References 18 publications

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“…In the wake of the landmark paper by Felgner et al, countless cationic lipids have been synthesized and studied [ 19 , 20 , 21 , 22 , 23 , 24 , 25 , 26 , 27 , 28 , 29 , 30 , 31 ]. In addition, numerous aspects of the formation and action of complexes of CLs with DNA and other nucleic acids (NAs) have been investigated [ 32 , 33 , 34 , 35 , 36 , 37 , 38 , 39 , 40 , 41 , 42 , 43 ].…”

Section: Introductionmentioning

confidence: 99%

Cationic Liposomes as Vectors for Nucleic Acid and Hydrophobic Drug Therapeutics

et al. 2021

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Cationic liposomes (CLs) are effective carriers of a variety of therapeutics. Their applications as vectors of nucleic acids (NAs), from long DNA and mRNA to short interfering RNA (siRNA), have been pursued for decades to realize the promise of gene therapy, with approvals of the siRNA therapeutic patisiran and two mRNA vaccines against COVID-19 as recent milestones. The long-term goal of developing optimized CL-based NA carriers for a broad range of medical applications requires a comprehensive understanding of the structure of these vectors and their interactions with cell membranes and components that lead to the release and activity of the NAs within the cell. Structure–activity relationships of lipids for CL-based NA and drug delivery must take into account that these lipids act not individually but as components of an assembly of many molecules. This review summarizes our current understanding of how the choice of the constituting lipids governs the structure of their CL–NA self-assemblies, which constitute distinct liquid crystalline phases, and the relation of these structures to their efficacy for delivery. In addition, we review progress toward CL–NA nanoparticles for targeted NA delivery in vivo and close with an outlook on CL-based carriers of hydrophobic drugs, which may eventually lead to combination therapies with NAs and drugs for cancer and other diseases.

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Section: Introductionmentioning

confidence: 99%

Cationic Liposomes as Vectors for Nucleic Acid and Hydrophobic Drug Therapeutics

et al. 2021

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“…[1][2][3][4][5][6][7][8] (ii) Many diagnostic applications with detection of specific base pairing (lab on the chip) require DNA adsorbed on a surface in a suitable way to be exposed to either complement and/or polymerase. [9][10][11] Compared with biological methods of gene transfer (like virus application), the use of lipid-DNA complexes is potentially less harmful to the organism.…”

Section: Introductionmentioning

confidence: 99%

DNA Alignment at Cationic Lipid Monolayers at the Air/Water Interface

et al. 2004

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The interaction of DNA with other DNA and with charged interfaces is of utmost importance for understanding nonviral transfection and DNA diagnostics with optimized chips. This can be studied in detail using Langmuir monolayers of cationic lipids as soft surface of a DNA containing subphase. The positional order of the lipid and the DNA sublattices can be studied by synchrotron X-ray diffraction under continuous variation of parameters such as molecular density or surface pressure. It is shown that DNA binding condenses the membrane surface, and the resulting structure is determined by the interplay of DNA-lipid and DNA-DNA interactions.

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“…Basically, cationic niosome formulations for gene delivery applications [17] contain a cationic lipid, which forms complexes with the negatively charged DNA and promotes the fusion with cell membranes [18], non-ionic surfactants to form stable emulsions and prevent particle aggregations [7] and "helper" components to enhance the biophysical properties [19]. Cationic lipids have three main functional domains: a hydrophilic head-group which enhances electrostatic interactions with the DNA and can be formed by quaternary ammoniums, amines, amino acids, lysine, guanidiniums and heterocycles [20], a hydrophobic domain usually composed of two saturated/unsaturated aliphatic chains [21] and a linker bond which influences in the stability, biodegradability, transfection efficiency and cytotoxicity of the cationic lipid [22]. This linker bond usually contains an ester, ester amide, carbamate, disulphide, urea or phosphate, among others [22].…”

Section: Introductionmentioning

confidence: 99%

Correlation between Biophysical Properties of Niosomes Elaborated with Chloroquine and Different Tensioactives and Their Transfection Efficiency

et al. 2021

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Lipid nanocarriers, such as niosomes, are considered attractive candidates for non-viral gene delivery due to their suitable biocompatibility and high versatility. In this work, we studied the influence of incorporating chloroquine in niosomes biophysical performance, as well as the effect of non-ionic surfactant composition and protocol of incorporation in their biophysical performance. An exhaustive comparative evaluation of three niosome formulations differing in these parameters was performed, which included the analysis of their thermal stability, rheological behavior, mean particle size, dispersity, zeta potential, morphology, membrane packing capacity, affinity to bind DNA, ability to release and protect the genetic material, buffering capacity and ability to escape from artificially synthesized lysosomes. Finally, in vitro biological studies were, also, performed in order to determine the compatibility of the formulations with biological systems, their transfection efficiency and transgene expression. Results revealed that the incorporation of chloroquine in niosome formulations improved their biophysical properties and the transfection efficiency, while the substitution of one of the non-ionic surfactants and the phase of addition resulted in less biophysical variations. Of note, the present work provides several biophysical parameters and characterization strategies that could be used as gold standard for gene therapy nanosystems evaluation.

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Optimization of Cationic Lipid Mediated Gene Transfer: Structure-Function, Physico-Chemical, and Cellular Studies

Cited by 14 publications

References 18 publications

Cationic Liposomes as Vectors for Nucleic Acid and Hydrophobic Drug Therapeutics

Cationic Liposomes as Vectors for Nucleic Acid and Hydrophobic Drug Therapeutics

DNA Alignment at Cationic Lipid Monolayers at the Air/Water Interface

Correlation between Biophysical Properties of Niosomes Elaborated with Chloroquine and Different Tensioactives and Their Transfection Efficiency

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