Peptide Nucleic Acids and Gene Editing: Perspectives on Structure and Repair.
Animals
Cystic Fibrosis
/ genetics
DNA
/ genetics
Disease Models, Animal
Gene Editing
/ methods
Gene Targeting
/ methods
Gene Transfer Techniques
Genetic Therapy
/ methods
Humans
Mice
Nanoparticles
/ chemistry
Nucleic Acid Conformation
Nucleic Acid Hybridization
Peptide Nucleic Acids
/ administration & dosage
Recombinational DNA Repair
beta-Thalassemia
/ genetics
PNA
cystic fibrosis
gene editing
nanoparticles
peptide nucleic acids
recombination
repair
structure
triplex
β-thalassemia
Journal
Molecules (Basel, Switzerland)
ISSN: 1420-3049
Titre abrégé: Molecules
Pays: Switzerland
ID NLM: 100964009
Informations de publication
Date de publication:
08 Feb 2020
08 Feb 2020
Historique:
received:
16
01
2020
revised:
05
02
2020
accepted:
06
02
2020
entrez:
13
2
2020
pubmed:
13
2
2020
medline:
24
11
2020
Statut:
epublish
Résumé
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.
Identifiants
pubmed: 32046275
pii: molecules25030735
doi: 10.3390/molecules25030735
pmc: PMC7037966
pii:
doi:
Substances chimiques
Peptide Nucleic Acids
0
DNA
9007-49-2
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : NHLBI NIH HHS
ID : UH3 HL147352
Pays : United States
Organisme : NIAID NIH HHS
ID : U01 AI145965
Pays : United States
Organisme : NHLBI NIH HHS
ID : F30 HL149185
Pays : United States
Organisme : NIGMS NIH HHS
ID : 5T32GM007223
Pays : United States
Organisme : NHLBI NIH HHS
ID : R01HL125892
Pays : United States
Organisme : NHLBI NIH HHS
ID : R01 HL139756
Pays : United States
Organisme : NHLBI NIH HHS
ID : F30HL149185
Pays : United States
Organisme : NIGMS NIH HHS
ID : T32 GM007205
Pays : United States
Organisme : NIGMS NIH HHS
ID : T32GM07205
Pays : United States
Organisme : NHLBI NIH HHS
ID : UG3 HL147352
Pays : United States
Organisme : NHLBI NIH HHS
ID : F30HL134252
Pays : United States
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