Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1161/CIRCRESAHA.124.325611 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
ABE-Mediated Cardiac Gene Silencing via Single AAVs Requires DNA Accessibility.
Circ Res
January 2025
School of Basic Medical Sciences, Institute of Cardiovascular Sciences, State Key Laboratory of Vascular Homeostasis and Remodeling, Peking University Health Science Center, Beijing, China. (Z.L., L.Y., Y.Y., J.L., Z.C., C.G., Y.G.).
Correction of DMD in human iPSC-derived cardiomyocytes by base-editing-induced exon skipping.
Mol Ther Methods Clin Dev
March 2023
Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program, The Ohio State University Wexner Medical Center, Columbus, OH 43210, USA.
Duchenne muscular dystrophy (DMD) is caused by mutations in the gene. Previously, we showed that adenine base editing (ABE) can efficiently correct a nonsense point mutation in a DMD mouse model. Here, we explored the feasibility of base-editing-mediated exon skipping as a therapeutic strategy for DMD using cardiomyocytes derived from human induced pluripotent stem cells (hiPSCs).
View Article and Find Full Text PDFBEON: A Functional Fluorescence Reporter for Quantification and Enrichment of Adenine Base-Editing Activity.
Mol Ther
July 2020
Department of Surgery, Davis Heart and Lung Research Institute, Biomedical Sciences Graduate Program, Biophysics Graduate Program, The Ohio State University Wexner Medical Center, Columbus, OH 43210, USA. Electronic address:
Adenine base editor (ABE) is a new generation of genome-editing technology through fusion of Cas9 nickase with an evolved E. coli TadA (TadA∗) and holds great promise as novel genome-editing therapeutics for treating genetic disorders. ABEs can directly convert A-T to G-C in specific genomic DNA targets without introducing double-strand breaks (DSBs).
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!