18352037. FUSION MOLECULES OF RATIONALLY-DESIGNED DNA-BINDING PROTEINS AND EFFECTOR DOMAINS simplified abstract (Duke University)
FUSION MOLECULES OF RATIONALLY-DESIGNED DNA-BINDING PROTEINS AND EFFECTOR DOMAINS
Organization Name
Inventor(s)
Michael G. Nicholson of Chapel Hill NC (US)
James Jefferson Smith of Morrisville NC (US)
FUSION MOLECULES OF RATIONALLY-DESIGNED DNA-BINDING PROTEINS AND EFFECTOR DOMAINS - A simplified explanation of the abstract
This abstract first appeared for US patent application 18352037 titled 'FUSION MOLECULES OF RATIONALLY-DESIGNED DNA-BINDING PROTEINS AND EFFECTOR DOMAINS
Simplified Explanation: The patent application describes targeted transcriptional effectors derived from meganucleases to regulate gene expression.
- These effectors consist of a DNA-binding domain from a meganuclease that binds to a specific target site and a transcription effector domain.
- The DNA-binding domain lacks endonuclease cleavage activity, making it safe for gene regulation without causing DNA damage.
Key Features and Innovation:
- Targeted transcriptional effectors derived from meganucleases
- DNA-binding domain without endonuclease activity
- Transcription effector domain for regulating gene expression
Potential Applications:
- Gene therapy
- Biotechnology research
- Precision medicine
Problems Solved:
- Safely regulate gene expression without causing DNA damage
- Target specific genes for therapeutic purposes
Benefits:
- Precise gene regulation
- Potential for targeted therapies
- Improved safety in gene editing
Commercial Applications: Potential commercial applications include gene therapy development, biotechnology research tools, and personalized medicine advancements.
Questions about Targeted Transcriptional Effectors: 1. How do targeted transcriptional effectors derived from meganucleases differ from traditional gene editing techniques? 2. What are the potential ethical considerations surrounding the use of targeted transcriptional effectors in gene therapy?
Original Abstract Submitted
Targeted transcriptional effectors (transcription activators and transcription repressors) derived from meganucleases are described. Also described are nucleic acids encoding same, and methods of using same to regulate gene expression. The targeted transcriptional effectors can comprise (i) a meganuclease DNA-binding domain lacking endonuclease cleavage activity that binds to a target recognition site; and (ii) a transcription effector domain.
