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Article ; Online: Reversible stabilization of DNA/PEI complexes by reducible click-linkage between DNA and polymer. A new polyplex concept for lowering polymer quantity.

Maze, Delphine / Pichon, Chantal / Midoux, Patrick

2023 Volume 30, Issue 12, Page(s) 783–791

Abstract: Nonviral transfection of mammalian cells can be performed with electrostatic complexes (polyplexes) between a plasmid DNA (pDNA) encoding a foreign gene and a cationic polymer. However, an excess of the cationic polymer is required for pDNA condensation ... ...

Abstract	Nonviral transfection of mammalian cells can be performed with electrostatic complexes (polyplexes) between a plasmid DNA (pDNA) encoding a foreign gene and a cationic polymer. However, an excess of the cationic polymer is required for pDNA condensation and polyplexes formation, which generate in vivo toxicity. Here, we present a new concept of polyplexes preparation aiming to reduce the polymer quantity. pDNA was functionalized with 3,6,9-trioxaundecan-1- {4 - [(2-chloroethyl) ethylamino)] - benzylamino}, 11-azide, and polyethyleneimine (lPEI) with reducible dibenzocyclooctyl (SS-DBCO) groups allowing azide-alkyne cycloaddition between pDNA and lPEI after condensation. The size of polyplexes with DBCO-SS-lPEI was smaller than with lPEI due to a stronger DNA condensation thanks to linkages between polymer and pDNA preventing dissociation until disulfide bridges reduction. In vitro transfection showed that the amount of DBCO-SS-lPEI leading to the most efficient polyplexes was three times lower than lPEI. As expected, toxicity in mice was significantly reduced upon intravenous injection of DBCO-SS-lPEI polyplexes at doses where the lPEI polyplexes killed mice. This is probably due to the high stability of the DBCO-SS-lPEI polyplexes which prevented their aggregation in the pulmonary capillaries. Overall, this new concept of polyplexes with DBCO-SS-lPEI offering the possibility of administering higher doses of polyplexes than lPEI and their ability to pass the pulmonary barrier could be favorably exploited for transfection of distant organs or tissues, such as tumors.
MeSH term(s)	Animals ; Mice ; Polymers ; Azides ; DNA/genetics ; Plasmids/genetics ; Transfection ; Polyethyleneimine ; Mammals/genetics
Chemical Substances	Polymers ; Azides ; DNA (9007-49-2) ; Polyethyleneimine (9002-98-6)
Language	English
Publishing date	2023-02-09
Publishing country	England
Document type	Journal Article
ZDB-ID	1191036-7
ISSN	1476-5462 ; 0969-7128
ISSN (online)	1476-5462
ISSN	0969-7128
DOI	10.1038/s41434-023-00386-1
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Article ; Online: CFTR and dystrophin encoding plasmids carrying both luciferase reporter gene, nuclear import specific sequences and triple helix sites.

Maze, Delphine / Girardin, Caroline / Benz, Nathalie / Montier, Tristan / Pichon, Chantal / Midoux, Patrick

Plasmid

2023 Volume 127, Page(s) 102686

Abstract: Duchenne Muscular Dystrophy and Cystic Fibrosis are two major monogenetic diseases which could be treated by non-viral gene therapy. For this purpose, plasmid DNA (pDNA) coding for the functional genes requires its equipment with signal molecules ... ...

Abstract	Duchenne Muscular Dystrophy and Cystic Fibrosis are two major monogenetic diseases which could be treated by non-viral gene therapy. For this purpose, plasmid DNA (pDNA) coding for the functional genes requires its equipment with signal molecules favouring its intracellular trafficking and delivery in the nucleus of the target cells. Here, two novel constructions of large pDNAs encoding the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) and full-length dystrophin (DYS) genes are reported. The expression of CFTR and DYS genes are driven respectively by the hCEF1 airway epithelial cells and spc5-12 muscle cells specific promoter. Those pDNAs encode also the luciferase reporter gene driven by the CMV promoter to evaluate gene delivery in animals by bioluminescence. In addition, oligopurine • oligopyrimidine sequences are inserted to enable equipment of pDNAs with peptides conjugated with a triple helix forming oligonucleotide (TFO). Furthermore, specific κB sequences are also inserted to promote their NFκB-mediated nuclear import. pDNA constructions are reported; transfection efficiency, tissue specific expression of CFTR and dystrophin in target cells, and triple helix formation are demonstrated. These plasmids are tools of interest to develop non-viral gene therapy of Cystic Fibrosis and Duchenne Muscular Dystrophy.
MeSH term(s)	Animals ; Active Transport, Cell Nucleus ; Cystic Fibrosis/genetics ; Cystic Fibrosis/therapy ; Cystic Fibrosis/metabolism ; Cystic Fibrosis Transmembrane Conductance Regulator/genetics ; Cystic Fibrosis Transmembrane Conductance Regulator/metabolism ; DNA ; Dystrophin/genetics ; Dystrophin/metabolism ; Genes, Reporter ; Muscular Dystrophy, Duchenne/genetics ; Muscular Dystrophy, Duchenne/therapy ; Plasmids/genetics
Chemical Substances	Cystic Fibrosis Transmembrane Conductance Regulator (126880-72-6) ; DNA (9007-49-2) ; Dystrophin
Language	English
Publishing date	2023-05-18
Publishing country	United States
Document type	Journal Article ; Research Support, Non-U.S. Gov't
ZDB-ID	282384-6
ISSN	1095-9890 ; 0147-619X
ISSN (online)	1095-9890
ISSN	0147-619X
DOI	10.1016/j.plasmid.2023.102686
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Article: mRNA Lipoplexes with Cationic and Ionizable α-Amino-lipophosphonates: Membrane Fusion, Transfection, mRNA Translation and Conformation.

Akhter, Sohail / Berchel, Mathieu / Jaffrès, Paul-Alain / Midoux, Patrick / Pichon, Chantal

Pharmaceutics

2022 Volume 14, Issue 3

Abstract: Cationic liposomes are attractive carriers for mRNA delivery. Here, mRNA lipoplexes (LX) were prepared with the cationic lipids α-aminolipophosphonate ( ...

Abstract	Cationic liposomes are attractive carriers for mRNA delivery. Here, mRNA lipoplexes (LX) were prepared with the cationic lipids α-aminolipophosphonate (
Language	English
Publishing date	2022-03-07
Publishing country	Switzerland
Document type	Journal Article
ZDB-ID	2527217-2
ISSN	1999-4923
ISSN	1999-4923
DOI	10.3390/pharmaceutics14030581
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Article: DNA minicircles as novel STAT3 decoy oligodeoxynucleotides endowed with anticancer activity in triple-negative breast cancer.

Casas, Geoffrey / Perche, Federico / Midoux, Patrick / Pichon, Chantal / Malinge, Jean-Marc

Molecular therapy. Nucleic acids

2022 Volume 29, Page(s) 162–175

Abstract: Decoy technology is a versatile and specific DNA oligonucleotide-based targeting strategy of pathogenic transcription factors (TFs). Chemical modifications of linear decoy oligonucleotides have been made to decrease nuclease sensitivity because of the ... ...

Abstract	Decoy technology is a versatile and specific DNA oligonucleotide-based targeting strategy of pathogenic transcription factors (TFs). Chemical modifications of linear decoy oligonucleotides have been made to decrease nuclease sensitivity because of the presence of free ends but at the cost of new limitations that affect their use as therapeutic drugs. Although a short DNA minicircle is a phosphodiester nucleic acid without free ends, its potential therapeutic activity as a TF decoy oligonucleotide has not yet been investigated. Here we describe the
Language	English
Publishing date	2022-06-22
Publishing country	United States
Document type	Journal Article
ZDB-ID	2662631-7
ISSN	2162-2531
ISSN	2162-2531
DOI	10.1016/j.omtn.2022.06.012
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Article: Intracellular Routing and Recognition of Lipid-Based mRNA Nanoparticles.

Delehedde, Christophe / Even, Luc / Midoux, Patrick / Pichon, Chantal / Perche, Federico

Pharmaceutics

2021 Volume 13, Issue 7

Abstract: Messenger RNA (mRNA) is being extensively used in gene therapy and vaccination due to its safety over DNA, in the following ways: its lack of integration risk, cytoplasmic expression, and transient expression compatible with fine regulations. However, ... ...

Abstract	Messenger RNA (mRNA) is being extensively used in gene therapy and vaccination due to its safety over DNA, in the following ways: its lack of integration risk, cytoplasmic expression, and transient expression compatible with fine regulations. However, clinical applications of mRNA are limited by its fast degradation by nucleases, and the activation of detrimental immune responses. Advances in mRNA applications, with the recent approval of COVID-19 vaccines, were fueled by optimization of the mRNA sequence and the development of mRNA delivery systems. Although delivery systems and mRNA sequence optimization have been abundantly reviewed, understanding of the intracellular processing of mRNA is mandatory to improve its applications. We will focus on lipid nanoparticles (LNPs) as they are the most advanced nanocarriers for the delivery of mRNA. Here, we will review how mRNA therapeutic potency can be affected by its interactions with cellular proteins and intracellular distribution.
Language	English
Publishing date	2021-06-24
Publishing country	Switzerland
Document type	Journal Article ; Review
ZDB-ID	2527217-2
ISSN	1999-4923
ISSN	1999-4923
DOI	10.3390/pharmaceutics13070945
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Article ; Online: CEBA: A new heterobifunctional reagent for plasmid DNA functionalization by click chemistry.

Gao, Haifei / Gonçalves, Cristine / Maze, Delphine / Pichon, Chantal / Midoux, Patrick

International journal of pharmaceutics

2021 Volume 601, Page(s) 120566

Abstract: Here, we report the synthesis of 3,6,9-trioxaundecan-1-{4-[(2-Chloroethyl)Ethylamino)]-Benzylamino},11-Azide (CEBA). CEBA alkylates the N7 of guanine of DNA thanks its chloroethyl group and can be coupled by a strain-promoted azide-alkyne cycloaddition ... ...

Abstract	Here, we report the synthesis of 3,6,9-trioxaundecan-1-{4-[(2-Chloroethyl)Ethylamino)]-Benzylamino},11-Azide (CEBA). CEBA alkylates the N7 of guanine of DNA thanks its chloroethyl group and can be coupled by a strain-promoted azide-alkyne cycloaddition to an alkynylated molecule. The optimization of the alkylation level of pDNA reveals that the expression of the encoded gene is preserved when it is randomly modified with at most 1 CEBA molecule per 150 bp. We show that the azido group of CEBA allows the linkage via click chemistry of CEBA-pDNA with a fluorophore or a peptide containing a dibenzocyclooctyne (DBCO) function. This new heterobifunctional reagent opens new ways to equip pDNA easily with signal molecules including peptides and nucleic acids without side products providing great interest for non-viral gene therapy.
MeSH term(s)	Alkynes ; Azides ; Click Chemistry ; DNA ; Plasmids/genetics
Chemical Substances	Alkynes ; Azides ; DNA (9007-49-2)
Language	English
Publishing date	2021-04-02
Publishing country	Netherlands
Document type	Journal Article
ZDB-ID	428962-6
ISSN	1873-3476 ; 0378-5173
ISSN (online)	1873-3476
ISSN	0378-5173
DOI	10.1016/j.ijpharm.2021.120566
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Zs.A 1409: Show issues

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Article ; Online: Impact of net charge, targeting ligand amount and mRNA modification on the uptake, intracellular routing and the transfection efficiency of mRNA lipopolyplexes in dendritic cells.

Delehedde, Christophe / Ciganek, Ivan / Rameix, Nathalie / Laroui, Nabila / Gonçalves, Cristine / Even, Luc / Midoux, Patrick / Pichon, Chantal

International journal of pharmaceutics

2023 Volume 647, Page(s) 123531

Abstract: Targeting mRNA formulations to achieve cell specificity is one of the challenges that must be tackled to mettle their therapeutic potential. Here, lipopolyplexes (LPR) bearing tri-mannose-lipid (TM) are used to target mannose receptor on dendritic cells. ...

Abstract	Targeting mRNA formulations to achieve cell specificity is one of the challenges that must be tackled to mettle their therapeutic potential. Here, lipopolyplexes (LPR) bearing tri-mannose-lipid (TM) are used to target mannose receptor on dendritic cells. We investigated the impact of the net charge and percentage of TM units on the binding, uptake, transfection efficiency (TE) and RNA sensors activation. Binding and uptake capacities of naked and targeted LPR increase with the percent of cationic lipid, but the latter are 2-fold more up taken by the cells. Cationic LPR bearing 5 % and 10 % TM were localized in acidic compartments in contrast to naked LPR and 2.5 % TM-LPR. The drawback is the dramatic decrease of TE as the number of TM-units increases. Cationic LPR bearing 5 % and 10 % TM strongly induced NF-κB and PKR phosphorylation at 6 h. Conversely, mTOR is less activated in line with their low TE. Those side effects are overcome by using 5-methoxyuridine mRNA resulting in an improved TE due to non-phosphorylation of NF-κB and PKR and mTOR activation. Our results point out that targeting DC via mannose receptor triggers a higher uptake of cationic LPRs and fast routing to acidic compartments, and that efficient TE requires low number of TM units use or modified mRNA to escape RNA sensors activation to enhance the translation.
MeSH term(s)	Ligands ; RNA, Messenger/genetics ; RNA, Messenger/metabolism ; Mannose Receptor ; NF-kappa B/genetics ; Transfection ; Lipids ; TOR Serine-Threonine Kinases/metabolism ; Dendritic Cells/metabolism ; Liposomes
Chemical Substances	Ligands ; RNA, Messenger ; Mannose Receptor ; NF-kappa B ; Lipids ; TOR Serine-Threonine Kinases (EC 2.7.11.1) ; Liposomes
Language	English
Publishing date	2023-10-18
Publishing country	Netherlands
Document type	Journal Article
ZDB-ID	428962-6
ISSN	1873-3476 ; 0378-5173
ISSN (online)	1873-3476
ISSN	0378-5173
DOI	10.1016/j.ijpharm.2023.123531
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Article ; Online: Neutral Block Copolymer Assisted Gene Delivery using Hydrodynamic Limb Vein Injection.

Guen, Yann Le / Delecourt, Gwendoline / Gall, Tony Le / Du, Haiqin / Illy, Nicolas / Huin, Cécile / Bennevault, Véronique / Midoux, Patrick / Montier, Tristan / Guégan, Philippe

Macromolecular bioscience

2024 , Page(s) e2300568

Abstract: Three different amphiphilic block copolymer families are synthesized to investigate new opportunities to enhance gene delivery via Hydrodynamic Limb Vein (HLV) injections. First a polyoxazoline-based family containing mostly one poly(2-methyl-2-oxazoline) ...

Abstract	Three different amphiphilic block copolymer families are synthesized to investigate new opportunities to enhance gene delivery via Hydrodynamic Limb Vein (HLV) injections. First a polyoxazoline-based family containing mostly one poly(2-methyl-2-oxazoline) (PMeOx) block and a second block POx with an ethyl (EtOx), isopropyl (iPrOx) or phenyl substituent (PhOx) is synthesized. Then an ABC poly(2-ethyl-2-oxazoline)-b-poly(2-n-propyl-2-oxazoline)-b-poly(2-methyl-2-oxazoline) triblock copolymer is synthesized, with a thermosensitive middle block. Finally, polyglycidol-b-polybutylenoxide-b-polyglycidol copolymers with various molar masses and amphiphilic balance are produced. The simple architecture of neutral amphiphilic triblock copolymer is not sufficient to obtain enhanced in vivo gene transfection. Double or triple amphiphilic neutral block copolymers are improving the in vivo transfection performances through HLV administration as far as a block having an lower critical solution temperature is incorporated in the vector. The molar mass of the copolymer does not seem to affect the vector performances in a significant manner.
Language	English
Publishing date	2024-03-21
Publishing country	Germany
Document type	Journal Article
ZDB-ID	2039130-4
ISSN	1616-5195 ; 1616-5187
ISSN (online)	1616-5195
ISSN	1616-5187
DOI	10.1002/mabi.202300568
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Article ; Online: CFTR and dystrophin encoding plasmids carrying both luciferase reporter gene, nuclear import specific sequences and triple helix sites

Maze, Delphine / Girardin, Caroline / Benz, Nathalie / Montier, Tristan / Pichon, Chantal / Midoux, Patrick

Plasmid. 2023 July, v. 127 p.102686-

2023

Abstract	Duchenne Muscular Dystrophy and Cystic Fibrosis are two major monogenetic diseases which could be treated by non-viral gene therapy. For this purpose, plasmid DNA (pDNA) coding for the functional genes requires its equipment with signal molecules favouring its intracellular trafficking and delivery in the nucleus of the target cells. Here, two novel constructions of large pDNAs encoding the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) and full-length dystrophin (DYS) genes are reported. The expression of CFTR and DYS genes are driven respectively by the hCEF1 airway epithelial cells and spc5–12 muscle cells specific promoter. Those pDNAs encode also the luciferase reporter gene driven by the CMV promoter to evaluate gene delivery in animals by bioluminescence. In addition, oligopurine • oligopyrimidine sequences are inserted to enable equipment of pDNAs with peptides conjugated with a triple helix forming oligonucleotide (TFO). Furthermore, specific κB sequences are also inserted to promote their NFκB-mediated nuclear import. pDNA constructions are reported; transfection efficiency, tissue specific expression of CFTR and dystrophin in target cells, and triple helix formation are demonstrated. These plasmids are tools of interest to develop non-viral gene therapy of Cystic Fibrosis and Duchenne Muscular Dystrophy.
Keywords	bioluminescence ; cystic fibrosis ; cystic fibrosis transmembrane conductance regulator ; dystrophin ; epithelium ; equipment ; gene therapy ; luciferase ; muscles ; muscular dystrophy ; oligonucleotides ; peptides ; physiological transport ; plasmids ; reporter genes ; transfection ; CFTR ; DNA constructs ; Triple helix ; Tissue specific promoter
Language	English
Dates of publication	2023-07
Publishing place	Elsevier Inc.
Document type	Article ; Online
ZDB-ID	282384-6
ISSN	1095-9890 ; 0147-619X
ISSN (online)	1095-9890
ISSN	0147-619X
DOI	10.1016/j.plasmid.2023.102686
Database	NAL-Catalogue (AGRICOLA)

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Zs.A 1736: Show issues

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Article ; Online: Selective attachment of a microtubule interacting peptide to plasmid DNA via a triplex forming oligonucleotide for transfection improvement.

Girardin, Caroline / Maze, Delphine / Gonçalves, Cristine / Le Guen, Yann Thierry / Pluchon, Kevin / Pichon, Chantal / Montier, Tristan / Midoux, Patrick

Gene therapy

2022 Volume 30, Issue 3-4, Page(s) 271–277

Abstract: In nonviral gene therapy approaches, the linkage of signal molecules to plasmid DNA (pDNA) is of interest for guiding its delivery to the nucleus. Here, we report its linkage to a peptide ( ... ...

Abstract	In nonviral gene therapy approaches, the linkage of signal molecules to plasmid DNA (pDNA) is of interest for guiding its delivery to the nucleus. Here, we report its linkage to a peptide (P
MeSH term(s)	Mice ; Animals ; Oligonucleotides/genetics ; Oligonucleotides/chemistry ; DNA/genetics ; Plasmids/genetics ; Transfection ; Microtubules/metabolism ; Peptides/genetics
Chemical Substances	Oligonucleotides ; DNA (9007-49-2) ; Peptides
Language	English
Publishing date	2022-07-07
Publishing country	England
Document type	Journal Article ; Research Support, Non-U.S. Gov't
ZDB-ID	1191036-7
ISSN	1476-5462 ; 0969-7128
ISSN (online)	1476-5462
ISSN	0969-7128
DOI	10.1038/s41434-022-00354-1
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