scholarly journals A universal discoidal nanoplatform for the intracellular delivery of PNAs

Nanoscale ◽  
2019 ◽  
Vol 11 (26) ◽  
pp. 12517-12529 ◽  
Author(s):  
Armin Tahmasbi Rad ◽  
Shipra Malik ◽  
Lin Yang ◽  
Tripat Kaur Oberoi-Khanuja ◽  
Mu-Ping Nieh ◽  
...  
Keyword(s):  
Nucleic Acids ◽  
Gene Editing ◽  
Peptide Nucleic Acids ◽  
Intracellular Delivery

Peptide nucleic acids (PNAs) have gained considerable attention due to their remarkable potential in gene editing and targeting-based strategies.

scholarly journals Phospholipid Conjugate for Intracellular Delivery of Peptide Nucleic Acids

2009 ◽  
Vol 20 (9) ◽  
pp. 1729-1736 ◽  
Author(s):  
Gang Shen ◽  
Huafeng Fang ◽  
Yinyin Song ◽  
Agata A. Bielska ◽  
Zhenghui Wang ◽  
...  
Keyword(s):  
Nucleic Acids ◽  
Peptide Nucleic Acids ◽  
Intracellular Delivery

scholarly journals Role of Cell-Penetrating Peptides in Intracellular Delivery of Peptide Nucleic Acids Targeting Hepadnaviral Replication

2017 ◽  
Vol 9 ◽  
pp. 162-169 ◽  
Author(s):  
Bénédicte Ndeboko ◽  
Narayan Ramamurthy ◽  
Guy Joseph Lemamy ◽  
Catherine Jamard ◽  
Peter E. Nielsen ◽  
...  
Keyword(s):  
Nucleic Acids ◽  
Peptide Nucleic Acids ◽  
Intracellular Delivery ◽  
Cell Penetrating Peptides ◽  
Cell Penetrating

scholarly journals Peptide Nucleic Acids and Gene Editing: Perspectives on Structure and Repair

Molecules ◽  
2020 ◽  
Vol 25 (3) ◽  
pp. 735 ◽  
Author(s):  
Nicholas G. Economos ◽  
Stanley Oyaghire ◽  
Elias Quijano ◽  
Adele S. Ricciardi ◽  
W. Mark Saltzman ◽  
...  
Keyword(s):  
Nucleic Acids ◽  
Gene Editing ◽  
Molecular Design ◽  
Peptide Nucleic Acids ◽  
Therapeutic Application ◽  
Monogenic Disorders ◽  
Structural Biochemistry ◽  
Nucleic Acid Structures ◽  
Target Effects

Unusual nucleic acid structures are salient triggers of endogenous repair and can occur in sequence-specific contexts. Peptide nucleic acids (PNAs) rely on these principles to achieve non-enzymatic gene editing. By forming high-affinity heterotriplex structures within the genome, PNAs have been used to correct multiple human disease-relevant mutations with low off-target effects. Advances in molecular design, chemical modification, and delivery have enabled systemic in vivo application of PNAs resulting in detectable editing in preclinical mouse models. In a model of β-thalassemia, treated animals demonstrated clinically relevant protein restoration and disease phenotype amelioration, suggesting a potential for curative therapeutic application of PNAs to monogenic disorders. This review discusses the rationale and advances of PNA technologies and their application to gene editing with an emphasis on structural biochemistry and repair.

Intracellular delivery of a peptide nucleic acid-based hybrid of an autophagy inducing peptide with a cell-penetrating peptide

2020 ◽  
Vol 18 (10) ◽  
pp. 1978-1986
Author(s):  
Yoshiyuki Hakata ◽  
Suzuka Ishikawa ◽  
Takashi Ohtsuki ◽  
Masaaki Miyazawa ◽  
Mizuki Kitamatsu
Keyword(s):  
Nucleic Acid ◽  
Nucleic Acids ◽  
Peptide Nucleic Acid ◽  
Peptide Nucleic Acids ◽  
Intracellular Delivery ◽  
Cell Penetrating Peptide ◽  
Cell Penetrating ◽  
A Cell

The conjugate of autophagy-inducing peptide with cell-penetrating peptide formed by hybridization between peptide nucleic acids was delivered into cell and induced effective autophagy.

scholarly journals An anionic, endosome-escaping polymer to potentiate intracellular delivery of cationic peptides, biomacromolecules, and nanoparticles

2019 ◽  
Vol 10 (1) ◽  
Author(s):  
Brian C. Evans ◽  
R. Brock Fletcher ◽  
Kameron V. Kilchrist ◽  
Eric A. Dailing ◽  
Alvin J. Mukalel ◽  
...  
Keyword(s):  
Nucleic Acids ◽  
Small Molecules ◽  
Gene Editing ◽  
Intracellular Delivery ◽  
Cationic Polymer ◽  
Peptide Delivery ◽  
Cationic Lipids ◽  
Cationic Peptides ◽  
Superior Efficacy ◽  
Intracellular Targets

Abstract Peptides and biologics provide unique opportunities to modulate intracellular targets not druggable by conventional small molecules. Most peptides and biologics are fused with cationic uptake moieties or formulated into nanoparticles to facilitate delivery, but these systems typically lack potency due to low uptake and/or entrapment and degradation in endolysosomal compartments. Because most delivery reagents comprise cationic lipids or polymers, there is a lack of reagents specifically optimized to deliver cationic cargo. Herein, we demonstrate the utility of the cytocompatible polymer poly(propylacrylic acid) (PPAA) to potentiate intracellular delivery of cationic biomacromolecules and nano-formulations. This approach demonstrates superior efficacy over all marketed peptide delivery reagents and enhances delivery of nucleic acids and gene editing ribonucleoproteins (RNPs) formulated with both commercially-available and our own custom-synthesized cationic polymer delivery reagents. These results demonstrate the broad potential of PPAA to serve as a platform reagent for the intracellular delivery of cationic cargo.

Growth Factor- and Adhesion Protein-Like Components of Fetal Calf Serum Can Significantly Enhance the Intracellular Delivery of Peptide Nucleic Acids

2011 ◽  
Vol 21 (4) ◽  
pp. 285-291 ◽  
Author(s):  
Johannes Oehlke ◽  
Angelika Ehrlich ◽  
Eberhard Krause ◽  
Stephan Pritz ◽  
Burkhard Wiesner ◽  
...  
Keyword(s):  
Growth Factor ◽  
Nucleic Acids ◽  
Fetal Calf Serum ◽  
Peptide Nucleic Acids ◽  
Calf Serum ◽  
Intracellular Delivery ◽  
Adhesion Protein

scholarly journals Peptide Nucleic Acids as a Tool for Site-Specific Gene Editing

Molecules ◽  
2018 ◽  
Vol 23 (3) ◽  
pp. 632 ◽  
Author(s):  
Adele Ricciardi ◽  
Elias Quijano ◽  
Rachael Putman ◽  
W. Saltzman ◽  
Peter Glazer
Keyword(s):  
Nucleic Acids ◽  
Gene Editing ◽  
Peptide Nucleic Acids ◽  
Specific Gene ◽  
Site Specific

Molecular insights on the dynamic stability of peptide nucleic acid functionalized carbon and boron nitride nanotubes

2021 ◽  
Vol 23 (1) ◽  
pp. 219-228
Author(s):  
Nabanita Saikia ◽  
Mohamed Taha ◽  
Ravindra Pandey
Keyword(s):  
Nucleic Acid ◽  
Boron Nitride ◽  
Nucleic Acids ◽  
Dynamic Stability ◽  
Peptide Nucleic Acid ◽  
Rational Design ◽  
Peptide Nucleic Acids ◽  
Boron Nitride Nanotubes ◽  
Molecular Hybrids ◽  
Single Wall Nanotubes

The rational design of self-assembled nanobio-molecular hybrids of peptide nucleic acids with single-wall nanotubes rely on understanding how biomolecules recognize and mediate intermolecular interactions with the nanomaterial's surface.

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