18551975. GENE CORRECTION FOR RAG2 DEFICIENCY IN HUMAN STEM CELLS simplified abstract (The Board of Trustees of the Leland Stanford Junior University)
Contents
- 1 GENE CORRECTION FOR RAG2 DEFICIENCY IN HUMAN STEM CELLS
- 1.1 Organization Name
- 1.2 Inventor(s)
- 1.3 GENE CORRECTION FOR RAG2 DEFICIENCY IN HUMAN STEM CELLS - A simplified explanation of the abstract
- 1.4 Simplified Explanation
- 1.5 Potential Applications
- 1.6 Problems Solved
- 1.7 Benefits
- 1.8 Potential Commercial Applications
- 1.9 Possible Prior Art
- 1.10 Unanswered Questions
- 1.11 Original Abstract Submitted
GENE CORRECTION FOR RAG2 DEFICIENCY IN HUMAN STEM CELLS
Organization Name
The Board of Trustees of the Leland Stanford Junior University
Inventor(s)
Mara Pavel-dinu of Stanford CA (US)
Matthew H. Porteus of Stanford CA (US)
GENE CORRECTION FOR RAG2 DEFICIENCY IN HUMAN STEM CELLS - A simplified explanation of the abstract
This abstract first appeared for US patent application 18551975 titled 'GENE CORRECTION FOR RAG2 DEFICIENCY IN HUMAN STEM CELLS
Simplified Explanation
The present disclosure provides methods and compositions for treating RAG2 deficiencies in subjects, comprising genetically modifying cells from the subjects ex vivo by integrating a functional, codon-optimized RAG 2 cDNA at the endogenous RAG2 locus.
- Genetically modifying cells ex vivo to treat RAG2 deficiencies in subjects
- Integrating a functional, codon-optimized RAG 2 cDNA at the endogenous RAG2 locus
Potential Applications
The technology could potentially be used in gene therapy to treat RAG2 deficiencies in patients.
Problems Solved
This technology addresses the issue of RAG2 deficiencies in subjects, providing a potential treatment option.
Benefits
- Offers a potential treatment for RAG2 deficiencies - Utilizes genetic modification to address the underlying cause of the condition
Potential Commercial Applications
- Gene therapy for RAG2 deficiencies: A New Treatment Approach
Possible Prior Art
There may be prior art related to gene therapy for genetic deficiencies, but specific examples related to RAG2 deficiencies may be limited.
Unanswered Questions
What are the potential long-term effects of this treatment?
Further research and clinical trials may be needed to determine the long-term effects of genetically modifying cells to treat RAG2 deficiencies.
How does this technology compare to existing treatments for RAG2 deficiencies?
Comparative studies with existing treatments, if any, could provide valuable insights into the effectiveness of this new approach.
Original Abstract Submitted
The present disclosure provides methods and compositions for treating RAG2 deficiencies in subjects, comprising genetically modifying cells from the subjects ex vivo by integrating a functional, codon-optimized RAG 2 cDNA at the endogenous RAG2 locus.
