18472881. RATIONALLY-DESIGNED MEGANUCLEASES WITH ALTERED SEQUENCE SPECIFICITY AND DNA-BINDING AFFINITY simplified abstract (Duke University)
Contents
RATIONALLY-DESIGNED MEGANUCLEASES WITH ALTERED SEQUENCE SPECIFICITY AND DNA-BINDING AFFINITY
Organization Name
Inventor(s)
James Jefferson Smith of Morrisville NC (US)
Homme W. Hellinga of Durham NC (US)
RATIONALLY-DESIGNED MEGANUCLEASES WITH ALTERED SEQUENCE SPECIFICITY AND DNA-BINDING AFFINITY - A simplified explanation of the abstract
This abstract first appeared for US patent application 18472881 titled 'RATIONALLY-DESIGNED MEGANUCLEASES WITH ALTERED SEQUENCE SPECIFICITY AND DNA-BINDING AFFINITY
Abstract: Rationally-designed LAGLIDADG meganucleases and methods of making such meganucleases are provided. In addition, methods are provided for using the meganucleases to generate recombinant cells and organisms having a desired DNA sequence inserted into a limited number of loci within the genome, as well as methods of gene therapy, for treatment of pathogenic infections, and for in vitro applications in diagnostics and research.
- Simplified Explanation:
This patent application discusses the creation of rationally-designed LAGLIDADG meganucleases and their use in inserting specific DNA sequences into limited loci in the genome for various applications.
- Key Features and Innovation:
- Development of rationally-designed LAGLIDADG meganucleases - Insertion of desired DNA sequences into specific loci in the genome - Applications in gene therapy, treatment of pathogenic infections, diagnostics, and research
- Potential Applications:
- Gene therapy - Treatment of pathogenic infections - Diagnostic applications - Research purposes
- Problems Solved:
- Precise insertion of DNA sequences into the genome - Limited number of loci targeted for insertion - Improved gene therapy techniques
- Benefits:
- Enhanced precision in genetic modifications - Targeted insertion of DNA sequences - Potential for more effective gene therapy treatments
- Commercial Applications:
Potential commercial applications include genetic engineering services, biotechnology research tools, and development of novel gene therapy treatments.
- Questions about LAGLIDADG Meganucleases:
1. How do rationally-designed LAGLIDADG meganucleases differ from other gene editing tools? 2. What are the specific challenges associated with using meganucleases for gene therapy applications?
Original Abstract Submitted
Rationally-designed LAGLIDADG meganucleases and methods of making such meganucleases are provided. In addition, methods are provided for using the meganucleases to generate recombinant cells and organisms having a desired DNA sequence inserted into a limited number of loci within the genome, as well as methods of gene therapy, for treatment of pathogenic infections, and for in vitro applications in diagnostics and research.
