CPC C12N 15/11 (2013.01) [C12N 9/22 (2013.01); C12N 9/96 (2013.01); C12N 15/111 (2013.01); C12N 15/907 (2013.01); C12N 2310/10 (2013.01); C12N 2310/20 (2017.05); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/346 (2013.01); C12N 2310/531 (2013.01); C12N 2320/51 (2013.01)] | 22 Claims |
1. A primary CD34+hematopoietic stem or progenitor cell (HSPC) comprising:
(a) a modified single guide RNA (sgRNA) comprising a nucleotide sequence that is complementary to a sequence within the HBB gene, wherein the modified sgRNA sequence is selected from the group consisting of SEQ ID NO: 6 and SEQ ID NO: 7;
(b) a Cas polypeptide, an mRNA encoding the Cas polypeptide, or a recombinant expression vector comprising a nucleotide sequence encoding the Cas polypeptide.
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