Anionic polymers for decreased toxicity and enhanced in vivo delivery of siRNA complexed with cationic liposomes

Schlegel, Anne; Largeau, Céline; Bigey, Pascal; Bessodes, Michel; Lebozec, Kristell; Scherman, Daniel; Escriou, Virginie

doi:10.1016/j.jconrel.2011.03.031

Cited by 70 publications

(76 citation statements)

References 27 publications

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“…However, PEG shielding is only partial since PEGylated polyplexes are still rapidly cleared compared to other well-known delivery systems (e.g., PEGylated liposomes), resulting in short blood circulation half-lives [9,10]. Alternatives to this approach consist of masking the net positive charge of the pre-formed complexes, by modification of with polyanions [11,12]. This results in the formation of negatively charged ternary complexes, which have shown decreased toxicity, improved in vivo stability and therapeutic effects in vivo [11].…”

Section: Introductionmentioning

confidence: 99%

Decationized polyplexes for gene delivery

Novo¹,

Mastrobattista²,

Nostrum³

et al. 2014

Expert Opinion on Drug Delivery

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Section: Introductionmentioning

confidence: 99%

Decationized polyplexes for gene delivery

Novo¹,

Mastrobattista²,

Nostrum³

et al. 2014

Expert Opinion on Drug Delivery

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“…Anionic polymer or its conjugate Cationic polymer or liposome Reference pDNA ᵞPGA Chitosan [66,67] pDNA ᵞPGA Dendrigraft poly-L-lysine [80] pDNA ᵞPGA PEI [81][82][83] pDNA ᵞPGA Protamine [75] pDNA αPGA DOTAP/Chol liposome [54] siRNA ᵞPGA Chitosan [73] siRNA ᵞPGA Dendrigraft poly-L-lysine [74] siRNA αPGA DOTAP/Chol liposome [77] siRNA αPGA DMAPAP/DOPE liposome [78,79] siRNA ᵞPGA PEI [76] pDNA Heparin Cationic glycopolymer (Tr4) [84] pDNA Heparin-PEI - [85] pDNA Alginic acid, Pectin Lipofectamine 2000 [24] pDNA: plasmid DNA, siRNA: small interfering RNA, ᵞPGA: poly-ᵞ-glutamic acid, αPGA: poly-α-glutamic acid, PEI: polyethylenimine, DMAPAP: 2-{3-[bis-(3-amino-propyl)-amino]-propylamino}-N-ditetradecyl carbamoyl methylacetamide, Chol: cholesterol, DOPE: 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine.…”

Section: Nucleic Acidmentioning

confidence: 99%

“…In addition, αPGA coating of 2-{3-[bis-(3-amino-propyl)-amino]-propylamino-Nditetradecyl carbamoyl methyl-acetamide (DMAPAP)/DOPE lipoplexes of siRNA ( Figure 3A) decreased in vitro and in vivo toxicities and enhanced siRNA delivery to the liver and lung after systemic injection [78,79]. Intravenous injection of γPGA is mainly accumulated in the spleen and liver in mice.…”

Section: Nucleic Acidmentioning

confidence: 99%

Progress in the development of Lipoplex and Polyplex modified with Anionic Polymer for efficient Gene Delivery

Hattori¹

2017

J Genet Med Gene Ther

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Nucleic acid-based therapy has become an increasingly important strategy for treating a variety of human diseases. In systemic therapy, a therapeutic gene must be delivered effi ciently to its target tissues without side effects. To deliver a therapeutic gene such as plasmid DNA (pDNA) or small interfering RNA (siRNA) to target tissues by systemic administration, cationic carriers such as cationic liposomes and polymers have been commonly used as a non-viral vector. However, the binary complex of therapeutic gene and cationic carrier must be stabilized in the blood circulation by avoiding agglutination with blood components, because electrostatic interactions between positively charged complexes and negatively charged erythrocytes can cause agglutination, and the agglutinates contribute to high entrapment of the therapeutic genes in the highly extended lung capillaries. One promising approach for overcoming this problem is modifi cation of the surface of cationic complexes with anionic biodegradable polymers such as hyaluronic acid, chondroitin sulfate, or polyglutamic acid. As another approach, we recently developed a sequential injection method of anionic polymer and cationic liposome/therapeutic gene complex (cationic lipoplex) for delivery of a therapeutic gene into the liver or liver metastasis. In this review, we describe recent advances in the delivery of therapeutic genes by lipid-and polymerbased carrier systems using anionic polymers.

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“…However, the use of viral vectors raise some safety concerns due to immune response [120]. Synthetic mimics that allow for the protection of the RNA in the extracellular environment but facilitate it's delivery within the cellular cytoplasm are therefore widely [121], lipid vesicles [122] and lipid-like structures (bolaamphiphiles [123]), anionic carriers [124], sugars [125], dendrimers [126], as well as gold [127] or silicon nanoparticles [128]. Gold nanoparticles can be used for vaccinations purposes [129].…”

Section: Delivery Of Multifunctional Rna and Dna Nanoparticlesmentioning

confidence: 99%

Triggering RNAi with multifunctional RNA nanoparticles and their delivery

Dao¹,

Viard²,

Martins³

et al. 2015

DNA and RNA Nanotechnology

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in the bloodstream, poor cellular uptake, and inefficient intracellular release. In an attempt to solve these issues, different types of RNAi therapeutic delivery strategies including multifunctional RNA nanoparticles are being developed. In this mini-review, we will briefly describe some of the current approaches.Keywords: RNA nanotechnology, RNA nanoparticles, RNA/DNA hybrids, RNA interference, siRNA, delivery IntroductionAccording to the Social Security Administration, average Americans that reach age 65 today most likely will live to be 84 years old [1]. However, with older age, the chances of contracting deadly diseases, such as cancer, increases dramatically. Some cancers (e.g. breast cancer) can be removed surgically but this does not guarantee that the disease will not return within a patient's lifetime. For other types of cancer (e.g. chronic lymphocytic leukemia), surgery may have very little effect (http://www. cancer.org). Other available treatments are chemo-and immunotherapies. However, these alternatives lack target specificity and cause severe toxic side effects affecting the growth of hair, nails, loss of appetite and blood cell count, just to name a few (http://www.cancer.org). Therefore, the advancements in biomedical technologies that provide safe and effective cancer treatment are in demand. Among the novel approaches is the recognition and use of specific intracellular RNA signatures (e.g. an overexpression of certain genes) that are especially important in detection and personalized treatments of cancers as well as viral infections, and autoimmune diseases [2][3][4]. The wide use of novel therapeutics based on target specific RNA-mediated gene silencing, called RNA interference or RNAi, will likely become the next breakthrough in cancer therapy. The first successful therapeutic knockdown of the endogenous gene, apolipoprotein B (ApoB), occurred in a 2004 study [5], only a few years after the original discovery of RNAi [6]. In 2010, DOI 10.1515/rnan-2015-0001 Received April 6, 2015 accepted May 6, 2015 Abstract: Proteins are considered to be the key players in structure, function, and metabolic regulation of our bodies. The mechanisms used in conventional therapies often rely on inhibition of proteins with small molecules, but another promising method to treat disease is by targeting the corresponding mRNAs. In 1998, Craig Mellow and Andrew Fire discovered dsRNA-mediated gene silencing via RNA interference or RNAi. This discovery introduced almost unlimited possibilities for new gene silencing methods, thus opening new doors to clinical medicine. RNAi is a biological process that inhibits gene expression by targeting the mRNA. RNAi-based therapeutics have several potential advantages (i) a priori ability to target any gene, (ii) relatively simple design process, (iii) sitespecificity, (iv) potency, and (v) a potentially safe and selective knockdown of the targeted cells. However, the problem lies within the formulation and delivery of RNAi therapeutics including rapid excretion, instab...

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Anionic polymers for decreased toxicity and enhanced in vivo delivery of siRNA complexed with cationic liposomes

Cited by 70 publications

References 27 publications

Decationized polyplexes for gene delivery

Decationized polyplexes for gene delivery

Progress in the development of Lipoplex and Polyplex modified with Anionic Polymer for efficient Gene Delivery

Triggering RNAi with multifunctional RNA nanoparticles and their delivery

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