Polymer-based non-viral vectors for gene therapy in the skin

Tortajada, Luz; Felip, Carles; Vicent, Marı́a J.

doi:10.1039/d1py01485d

Cited by 10 publications

(5 citation statements)

References 131 publications

(147 reference statements)

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“…[4][5][6] Non-viral gene vectors, including liposomes, polymeric nanoparticles and inorganic nanoparticle, have attracted significant attention for gene delivery due to their biocompatibility and ability to encapsulate a wide range of therapeutic molecules. [7][8][9][10] Nonetheless, there are limitations in efficient gene loading and expression due to their low serum stability, limited load capacity and uncontrollable release, [11] prompting the development of nanocomposite delivery platform like functionalized mesoporous silicon. [12,13] Mesoporous silica nanoparticles (MSNs) are extensively characterized and widely explored as potential vehicles for drug or gene due to low toxicity, large surface area, ordered pore structure, super high specific pore volume, tunable pore size, and easy surface modifiability.…”

Section: Introductionmentioning

confidence: 99%

“…However, effective gene delivery necessitates a carrier capable of safeguarding therapeutic genes from degradation, facilitating cellular uptake, and promoting their release within cells [4–6] . Non‐viral gene vectors, including liposomes, polymeric nanoparticles and inorganic nanoparticle, have attracted significant attention for gene delivery due to their biocompatibility and ability to encapsulate a wide range of therapeutic molecules [7–10] . Nonetheless, there are limitations in efficient gene loading and expression due to their low serum stability, limited load capacity and uncontrollable release, [11] prompting the development of nanocomposite delivery platform like functionalized mesoporous silicon [12,13] …”

Section: Introductionmentioning

confidence: 99%

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Temperature/GSH Dual Responsive Fluorinated Mesoporous Silica‐Coated Au NRs for Serum‐Resistant Gene Delivery**

Sun,

Fu,

Zhang

et al. 2024

ChemistrySelect

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Mesoporous silica nanoparticles (MSN) are emerging as one of the most appealing candidates for gene carriers. Nevertheless, one major obstacle for their clinical application is the lack of efficient controlled release and gene delivery. In this study, a mesoporous silica‐modified gold nanorods (Au NRs) controlled release platform was synthesized. Then a thermosensitive copolymer was grafted to the surface of release platform, to impart the switching response of carrier under the photothermal of Au NRs. Fluorination were modified on the surface of MSN through a disulfide bond to achieve glutathione (GSH) response release and improve serum‐resistant ability. This gene carrier, with notable DNA condensation ability and negligible cytotoxicity, could be easily internalized into cells, enhanced green fluorescent protein expression in the presence of serum under laser irradiation. This work inspired us to solve the problem of traditional controllable sustained release and realize the performance of intracellular switch response to gene sustained release.

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Temperature/GSH Dual Responsive Fluorinated Mesoporous Silica‐Coated Au NRs for Serum‐Resistant Gene Delivery**

Sun,

Fu,

Zhang

et al. 2024

ChemistrySelect

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“…The most effective solution to these intrinsic problems of nucleic acid drugs is to use nanoarchitectures as drug carriers (Figure 2). [14][15][16][17][18][19][20][21][22][23][24][25] In these carriers, all the requirements for smart drug delivery (size of nanomedicine, its shape, membrane penetration, selective binding to target cells, and many other Encapsulation of nucleic acid drugs by nanoarchitectures. In these nanostructures, otherwise unstable DNA and RNA are protected from enzymatic digestion.…”

Section: Introductionmentioning

confidence: 99%

“…The most effective solution to these intrinsic problems of nucleic acid drugs is to use nanoarchitectures as drug carriers (Figure 2). [14–25] In these carriers, all the requirements for smart drug delivery (size of nanomedicine, its shape, membrane penetration, selective binding to target cells, and many other features) can be fulfilled in terms of elegantly designed three‐dimensional nanostructures. For example, nucleic acid drugs are tenderly nested in them, and protected from hydrolytic enzymes and other intracellular degradations until reaching target site.…”

Section: Introductionmentioning

confidence: 99%

Nanoarchitectures to Deliver Nucleic Acid Drugs to Disease Sites

Komiyama

Sumaoka

2023

ChemNanoMat

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Therapeutic nucleic acid drugs (antisense DNA, siRNA, DNAzyme, and others) have been widely employed to regulate the expression of a disease‐causing gene. For the correction of a gene, the CRISPR/Cas9 system is available. Advantageously, they can be straightforwardly designed in terms of the Watson‐Crick rule. In order to deliver these nucleic acid drugs to a target place in our body (organs and cells) at desired timing, various nanoarchitectures have been elegantly designed. Their primary roles are protection of drugs from degradation, increase of cell‐membrane permeability, and spatiotemporal control of delivery. Encapsulated nucleic acid drugs are released by decomposing the nanostructures with either external stimuli or intracellular signals. Furthermore, therapeutic efficiency is enhanced by simultaneously delivering multiple types of nucleic acid drugs for their cooperation. Composites of nanoarchitectures and therapeutic nucleic acid drugs are highly promising for future applications.

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“…[2][3][4] By contrast, various forms of nonviral vectors are widely developed and applied in biomedical research because of their low immunogenicity, high biocompatibility, simplicity in structural pattern, ease of size adjustment, practicality in designing various active sites, and low cost of production. [5,6] The most widely studied nonviral vectors are liposomes, inorganic nanoparticles, polymers, and dendrimers. [7] Liposomes are small spherical vesicles from cholesterol and phospholipids characterized with the surface that can be modified using glycolipids or sialic acid.…”

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confidence: 99%

Polymeric Dendrimers as Nanocarrier Vectors for Neurotheranostics

Zha

Liu

et al. 2022

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immune responses, activating cellular machinery causing cell stress, cytotoxicity, lack of selectivity, increasing the likelihood of replication competence, and the constrained structure of insert-size, which can limit their biological and medical applications. [2][3][4] By contrast, various forms of nonviral vectors are widely developed and applied in biomedical research because of their low immunogenicity, high biocompatibility, simplicity in structural pattern, ease of size adjustment, practicality in designing various active sites, and low cost of production. [5,6] The most widely studied nonviral vectors are liposomes, inorganic nanoparticles, polymers, and dendrimers. [7] Liposomes are small spherical vesicles from cholesterol and phospholipids characterized with the surface that can be modified using glycolipids or sialic acid. Yet, the unmodified forms of liposomes are structurally unstable and could easily be eliminated by means of body circulation. [8] In this situation, stable inorganic nanoparticles, such as calcium phosphate (Ca 3 (PO 4 ) 2 ), gold (Au), iron oxide (Fe 3 O 4 ), and lanthanide-based nanoparticles are widely used, mainly due to their good hydrophilicity and excellent biocompatibility. [9][10][11] Depending on their chemical core composition, these inorganic nanoparticles display different properties and efficiencies. Lately, inorganic nanoparticles have been developed as effective drug delivery systems and used in many diagnostic techniques. [12] In addition, polymers are widely used as nonviral vectors as well. [13][14][15] Polymers are biodegradable nanomaterials with stable and easily modifiable 3D structures. Their uncomplicated synthesis process allows for large-scale production and safe clinical use compared to inorganic nanoparticles. [16] Notably, dendrimers are hyperbranched polymers with a well-defined chemical structure: a core at the center occupied by array of convergent reactive chain-ends and surface that can easily be modified. [17] The chemistry of dendrimers was first described by Buhleier et al. in 1978. [18] In 1985, Tomalia et al. reported a newer kind of dendrimers based on the mixture of amines and amides, termed polyamidoamine (PAMAM) dendrimers. [19] Dendrimers are intermeshed synthetic organic macromolecules with 3D architecture composition and abundant terminal functional groups. They are well-defined monodispersity-sized materials with extraordinary membrane interaction properties Dendrimers are polymers with well-defined 3D branched structures that are vastly utilized in various neurotheranostics and biomedical applications, particularly as nanocarrier vectors. Imaging agents can be loaded into dendrimers to improve the accuracy of diagnostic imaging processes. Likewise, combining pharmaceutical agents and anticancer drugs with dendrimers can enhance their solubility, biocompatibility, and efficiency. Practically, by modifying ligands on the surface of dendrimers, effective therapeutic and diagnostic platforms can be constructed and implemented for target...

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Polymer-based non-viral vectors for gene therapy in the skin

Abstract: Gene therapy has emerged as a versatile technique with the potential to treat a range of human diseases; however, examples of the topical application of gene therapy as a treatment...

Cited by 10 publications

References 131 publications

Temperature/GSH Dual Responsive Fluorinated Mesoporous Silica‐Coated Au NRs for Serum‐Resistant Gene Delivery**

Temperature/GSH Dual Responsive Fluorinated Mesoporous Silica‐Coated Au NRs for Serum‐Resistant Gene Delivery**

Nanoarchitectures to Deliver Nucleic Acid Drugs to Disease Sites

Polymeric Dendrimers as Nanocarrier Vectors for Neurotheranostics

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