GENE CORRECTION OF SCID-RELATED GENES IN HEMATOPOIETIC STEM AND PROGENITOR CELLS

摘要

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. The present invention describes compositions and methods for use in gene therapy and genome engineering. Specifically, the methods and compositions described relate to targeted insertion of a transgene (donor) including protein encoding sequence, for example a protein that is lacking or deficient in a subject with a SCID.