Peptide nucleic acid delivered by nanoparticles as a gene editing tool: Methods of design, preparation and evaluation
Eltaweel, N., Amr, K., ElKamah, G., Ahmed, N., Ibrahim, I. (2022). Peptide nucleic acid delivered by nanoparticles as a gene editing tool: Methods of design, preparation and evaluation. EKB Journal Management System, 2(2), 139-147. doi: 10.21608/aijpms.2022.122686.1113
Noha H Eltaweel; Khalda S Amr; Ghada ElKamah; Nermeen E Ahmed; Iman H Ibrahim. "Peptide nucleic acid delivered by nanoparticles as a gene editing tool: Methods of design, preparation and evaluation". EKB Journal Management System, 2, 2, 2022, 139-147. doi: 10.21608/aijpms.2022.122686.1113
Eltaweel, N., Amr, K., ElKamah, G., Ahmed, N., Ibrahim, I. (2022). 'Peptide nucleic acid delivered by nanoparticles as a gene editing tool: Methods of design, preparation and evaluation', EKB Journal Management System, 2(2), pp. 139-147. doi: 10.21608/aijpms.2022.122686.1113
Eltaweel, N., Amr, K., ElKamah, G., Ahmed, N., Ibrahim, I. Peptide nucleic acid delivered by nanoparticles as a gene editing tool: Methods of design, preparation and evaluation. EKB Journal Management System, 2022; 2(2): 139-147. doi: 10.21608/aijpms.2022.122686.1113

Peptide nucleic acid delivered by nanoparticles as a gene editing tool: Methods of design, preparation and evaluation

Azhar International Journal of Pharmaceutical and Medical Sciences
Article 14, Volume 2, Issue 2, June 2022, Page 139-147  XML PDF (401.55 K)
Document Type: Original research articles
DOI: 10.21608/aijpms.2022.122686.1113
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Authors
Noha H Eltaweel email orcid 1; Khalda S Amrorcid 1; Ghada ElKamahorcid 2; Nermeen E Ahmedorcid 3; Iman H Ibrahimorcid 4
1Deparment of Medical Molecular Genetics, National Research Centre, Cairo, Egypt
2Department of Clinical Genetics, National Research Centre, Egypt
3Department of Oro-Dental Genetics, National Research Centre, Cairo, Egypt
4Department of Biochemistry and Molecular Biology, Faculty of Pharmacy (Girls), Al-Azhar University, Cairo, Egypt
Abstract
Synthetic nucleic acids could be designed in sequence-specific structure to trigger endogenous repair systems. Depending on this concept, peptide nucleic acids (or PNAs) could achieve non-enzymatic gene editing i.e. without the use of nucleases unlike CRISPR and TALENs. PNAs could bind to the DNA to form highly specific hetero-triplex structures, so PNAs have been used severally in the last decades to induce correction of different human disease-causing mutations with low off-targets. Systemic in-vivo and in-utero application of PNAs had been enabled thanks to the advances in their chemical structure, design and delivery resulting in considerable preclinical editing in mouse models. Treated animal models engineered with a human beta globin gene (HBB gene carrying a β-thalassemia mutation, showed clinically considerable protein expression, suggesting that PNAs could have a curative potential for genetic disorders. The complete methods of design, preparation and evaluation of the PNA/DNA nanoparticles formulation will be discussed in details in addition to its use in cells treatment aiming for paving the road for interested scientists in gene editing techniques for treating different single gene disorders.
Keywords
peptide nucleic acids; PNA; gene editing; triplex; nanoparticles; PLGA
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