GENE CORRECTION OF SCID-RELATED GENES IN HEMATOPOIETIC STEM AND PROGENITOR CELLS
First Claim
Patent Images
1. A nuclease comprising a DNA-binding domain and cleavage domain, wherein the DNA-binding domain binds to a target site in a severe combined immunodeficiency (SCID)-related gene, wherein the target site is intron 1 of an endogenous IL2RG gene or intron 1 or 2 of an endogenous RAG gene.
2 Assignments
0 Petitions
Accused Products
Abstract
The present disclosure is in the field of genome engineering, particularly targeted integration of a functional SCID-related genes (e.g., IL2RG, RAG1 and/or RAG2 gene) into the genome of a cell for provision of proteins lacking or deficient in SCID.
18 Citations
19 Claims
- 1. A nuclease comprising a DNA-binding domain and cleavage domain, wherein the DNA-binding domain binds to a target site in a severe combined immunodeficiency (SCID)-related gene, wherein the target site is intron 1 of an endogenous IL2RG gene or intron 1 or 2 of an endogenous RAG gene.
Specification