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

doi:10.1016/j.omtn.2022.06.012

Cited by 7 publications

(1 citation statement)

References 49 publications

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“…A number of derivative vectors from pEPI-1 were constructed and were used to test the S/MAR technology in different settings: (i) episomal expression in mouse liver [ 14 ], (ii) elimination of harmful sequences which increases safety, such as in the minicircle [ 15 ] in vitro and in vivo [ 16 ], in a pre-clinical model [ 17 ], in the pEPIto vector [ 18 ] and in an in vivo study [ 19 ], (iii) efficient episomal gene transfer suitable for liver-directed studies with the pFAR-S/MAR vector [ 20 ], (iv) genetic modification of pluripotent stem cells with nanovectors [ 21 ], (v) generation of chimeric antigen receptor (CAR)-T cells [ 22 ], and (viii) enhancement of viral episomes when combined with the S/MAR element [ 23 ]. Evidently, the S/MAR element is a bona fide modulator of chromatin that confers non-integration as well as mitotic stability to the plasmid derivatives of pEPI-1, in which it is included.…”

Section: Introductionmentioning

confidence: 99%

Non-Viral Episomal Vector Mediates Efficient Gene Transfer of the β-Globin Gene into K562 and Human Haematopoietic Progenitor Cells

Lazaris,

Simantirakis,

Stavrou

et al. 2023

Genes

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β-Thalassemia is a subgroup of inherited blood disorders associated with mild to severe anemia with few and limited conventional therapy options. Lately, lentiviral vector-based gene therapy has been successfully applied for disease treatment. However, the current development of non-viral episomal vectors (EV), non-integrating and non-coding for viral proteins, may be helpful in generating valid alternatives to viral vectors. We constructed a non-viral, episomal vector pEPβ-globin for the physiological β-globin gene based on two human chromosomal elements: the scaffold or matrix attachment region (S/MAR), allowing for long nuclear retention and non-integration and the β-globin replication initiation region (IR), allowing for enhancement of replication and establishment. After nucleofections into K562 cells with a transfection efficiency of 24.62 ± 7.7%, the vector induces stable transfection and is detected in long-term cultures as a non-integrating, circular episome expressing the β-globin gene efficiently. Transfections into CD34+ cells demonstrate an average efficiency of 15.57 ± 11.64%. In the colony-forming cell assay, fluorescent colonies are 92.21%, which is comparable to those transfected with vector pEP-IR at 92.68%. Additionally, fluorescent colonies produce β-globin mRNA at a physiologically 3-fold higher level than the corresponding non-transfected cells. Vector pEPβ-globin provides the basis for the development of therapeutic EV for gene therapy of β-thalassemias.

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Section: Introductionmentioning

confidence: 99%

Non-Viral Episomal Vector Mediates Efficient Gene Transfer of the β-Globin Gene into K562 and Human Haematopoietic Progenitor Cells

Lazaris,

Simantirakis,

Stavrou

et al. 2023

Genes

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Enhancing the Features of DNA Mimic Foldamers for Structural Investigations

Corvaglia,

Wu,

Deepak

et al. 2024

Chemistry A European J

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DNA mimic foldamers based on aromatic oligoamide helices bearing anionic phosphonate side chains have been shown to bind to DNA‐binding proteins sometimes orders of magnitude better than DNA itself. Here, we introduce new features in the DNA mimic foldamers to facilitate structural investigations of their interactions with proteins. Thirteen new foldamer sequences have been synthesized and characterized using NMR, circular dichroism, molecular modeling, and X‐ray crystallography. The results show that foldamer helix handedness can be quantitatively biased by means of a single stereogenic center, that the foldamer structure can be made C2‐symmetrical as in palindromic B‐DNA sequences, and that associations between foldamer helices can be promoted utilizing dedicated C‐terminal residues that act as sticky ends in B‐DNA structures.

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The future of genetic medicines delivered via targeted lipid nanoparticles to leukocytes

Tarab-Ravski,

Stotsky-Oterin,

Elisha

et al. 2024

Journal of Controlled Release

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DNA minicircles as novel STAT3 decoy oligodeoxynucleotides endowed with anticancer activity in triple-negative breast cancer

Cited by 7 publications

References 49 publications

Non-Viral Episomal Vector Mediates Efficient Gene Transfer of the β-Globin Gene into K562 and Human Haematopoietic Progenitor Cells

Non-Viral Episomal Vector Mediates Efficient Gene Transfer of the β-Globin Gene into K562 and Human Haematopoietic Progenitor Cells

Enhancing the Features of DNA Mimic Foldamers for Structural Investigations

The future of genetic medicines delivered via targeted lipid nanoparticles to leukocytes

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