18007439. GENE CORRECTION FOR SCID-X1 IN LONG-TERM HEMATOPOIETIC STEM CELLS simplified abstract (THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY)
Contents
- 1 GENE CORRECTION FOR SCID-X1 IN LONG-TERM HEMATOPOIETIC STEM CELLS
- 1.1 Organization Name
- 1.2 Inventor(s)
- 1.3 GENE CORRECTION FOR SCID-X1 IN LONG-TERM HEMATOPOIETIC 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 Original Abstract Submitted
GENE CORRECTION FOR SCID-X1 IN LONG-TERM HEMATOPOIETIC 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 SCID-X1 IN LONG-TERM HEMATOPOIETIC STEM CELLS - A simplified explanation of the abstract
This abstract first appeared for US patent application 18007439 titled 'GENE CORRECTION FOR SCID-X1 IN LONG-TERM HEMATOPOIETIC STEM CELLS
Simplified Explanation
The present disclosure involves methods and compositions for treating SCID-X1 in subjects by genetically modifying cells ex vivo with a full-length, codon-optimized IL2RG cDNA at the endogenous IL2RG locus.
- Genetically modifying cells from subjects ex vivo
- Integrating a full-length, codon-optimized IL2RG cDNA at the endogenous IL2RG locus
Potential Applications
The technology could potentially be used in the treatment of SCID-X1 in subjects by correcting the genetic mutation responsible for the condition.
Problems Solved
This technology addresses the genetic mutation that causes SCID-X1, providing a potential treatment option for affected individuals.
Benefits
- Offers a potential treatment for SCID-X1 - Targets the root cause of the condition by correcting the genetic mutation
Potential Commercial Applications
The technology could have commercial applications in the field of gene therapy for SCID-X1 and other genetic disorders.
Possible Prior Art
One possible prior art could be the use of gene therapy in treating genetic disorders, including SCID-X1.
Unanswered Questions
How does this technology compare to existing treatments for SCID-X1?
This article does not provide a direct comparison to existing treatments for SCID-X1. Further research or clinical trials may be needed to evaluate the efficacy and safety of this new approach.
What are the potential long-term effects of genetically modifying cells in this manner?
The article does not address the potential long-term effects of genetically modifying cells with a full-length, codon-optimized IL2RG cDNA. Additional studies may be required to assess the long-term safety and efficacy of this treatment approach.
Original Abstract Submitted
The present disclosure provides methods and compositions for treating SCID-X1 in subjects, comprising genetically modifying cells from the subjects ex vivo by integrating a full-length, codon-optimized IL2RG cDNA at the endogenous IL2RG locus.
