Genome editing with site-specific nucleases (SSNs) may be effective for gene therapy, as SSNs can modify target genes. However, the main limitation of genome editing for clinical use is off-target effects by excess amounts of SSNs within cells. Therefore, a controlled delivery system for SSNs is necessary. Previously we have reported on a zinc finger nuclease (ZFN) delivery system, which combined DNA aptamers against FokI nuclease domain (FokI) and nanoneedles. Here, we describe how DNA aptamers against FokI were selected and characterized for genome editing applications.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1007/978-1-4939-8799-3_12 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Advances in Functional Nucleic Acid SERS Sensing Strategies.
ACS Sens
January 2025
Center for Biomedical-photonics and Molecular Imaging, Advanced Diagnostic-Therapy Technology and Equipment Key Laboratory of Higher Education Institutions in Shaanxi Province, School of Life Science and Technology, Xidian University, Xi'an, Shaanxi 710126, China.
Functional nucleic acids constitute a distinct category of nucleic acids that diverge from conventional nucleic acid amplification methodologies. They are capable of forming intricate hybrid structures through Hoogsteen and reverse Hoogsteen hydrogen bonding interactions between double-stranded and single-stranded DNA, thereby broadening the spectrum of DNA interactions. In recent years, functional DNA/RNA-based surface-enhanced Raman spectroscopy (SERS) has emerged as a potent platform capable of ultrasensitive and multiplexed detection of a variety of analytes of interest.
View Article and Find Full Text PDFATLAS-seq: a microfluidic single-cell TCR screen for antigen-reactive TCRs.
Nat Commun
January 2025
Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Discovering antigen-reactive T cell receptors (TCRs) is central to developing effective engineered T cell immunotherapies. However, the conventional technologies for isolating antigen-reactive TCRs (i.e.
View Article and Find Full Text PDFMultifunctional DNA nanomaterials: a new frontier in rheumatoid arthritis diagnosis and treatment.
Nanoscale
January 2025
State Key Laboratory of Oral Diseases & National Center for Stomatology & National Clinical Research Center for Oral Diseases, West China Hospital of Stomatology, Sichuan University, Chengdu 610041, Sichuan, China.
Rheumatoid arthritis (RA) remains a challenging autoimmune disease due to its complex and heterogeneous pathophysiology, which complicates therapeutic and diagnostic efforts. Advances in DNA nanotechnology have introduced DNA nanomaterials as promising tools to overcome these barriers. This review focuses on three primary categories of DNA nanomaterials applied in RA: DNA nanostructures, DNA aptamers, and DNA-modified nanoparticles.
View Article and Find Full Text PDFRNA Diagnostics and Therapeutics: A Comprehensive Review.
RNA Biol
January 2025
Department of Biotechnology, Manipal Institute of Technology (MIT), Manipal Academy of Higher Education (MAHE), Manipal, Karnataka 576104, India.
RNA-focused therapy and diagnostics have been making waves in molecular biology due to the advantages RNA has over DNA; for instance, the ability of RNA to target nearly any genetic component in the cell is a big step in treating disorders. Moreover, RNA-based diagnosis of diseases is only becoming increasingly popular, especially after the COVID-19 pandemic, which brought up the need for cost-effective and efficient diagnosing kits for the vast majority. RNA-based techniques also have close to no risk of genotoxicity and can efficiently target undruggable regions of the cell.
View Article and Find Full Text PDFDevelopment of dual aptamers-functionalized c-MET PROTAC degraders for targeted therapy of osteosarcoma.
Theranostics
January 2025
Department of Systems Biology, School of Life Sciences, Southern University of Science and Technology, Shenzhen 518055, China.
Osteosarcoma (OS) is the most common bone malignancy. c-MET is recognized as a therapeutic target. However, traditional c-MET inhibitors show compromised efficacy due to the acquired resistance and side effects.
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!