Expression of human Foxp3 in gene editing T cells

摘要

The aspect of the invention described herein relates to the generation of Foxp3 in a human primary lymphoid or under control by targeting Foxp3 cDNA (e.g., the full length of Foxp3 cDNA optimized for human codon) to the FOXP3 locus or non Foxp3 locus.The compositions and materials described herein provide specificity for control of mice, non human primates, or fofox3 genes in humans through cripr / CAS.Using a guide RNA sequence, the FOXP3 locus, aavs1 locus, and other candidate loci are targeted to perform gene regulation through CRISPR / CAS and provide gene delivery cassettes for gene modification based on homologous recombination repair.The alternative compositions described herein can be delivered in the form of ribonucleoprotein (RNP) and may be used to target Foxp3 of human and / or non human primates.The reagent is composed of a novel guide RNA sequence, which can be used to generate an on target cleavage at high frequencies by combining with a novel gene delivery cassette and a CAS protein comprising Foxp3 cDNA and / or other CIS configuration gene products.