US 11,851,652 B2
Compositions comprising chemically modified guide RNAs for CRISPR/Cas-mediated editing of HBB
Matthew H. Porteus, Stanford, CA (US); Ayal Hendel, Stanford, CA (US); Joe Clark, Stanford, CA (US); Rasmus O. Bak, Stanford, CA (US); Daniel E. Ryan, Santa Clara, CA (US); Douglas J. Dellinger, Santa Clara, CA (US); Robert Kaiser, Santa Clara, CA (US); and Joel Myerson, Santa Clara, CA (US)
Assigned to THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR, Stanford, CA (US); and UNIVERSITY and AGILENT TECHNOLOGIES, INC., Santa Clara, CA (US)
Filed by The Board of Trustees of the Leland Stanford Junior University, Stanford, CA (US)
Filed on Mar. 14, 2022, as Appl. No. 17/694,361.
Application 17/694,361 is a continuation of application No. 15/724,073, filed on Oct. 3, 2017, granted, now 11,306,309.
Application 15/724,073 is a continuation of application No. PCT/US2016/026028, filed on Apr. 5, 2016.
Claims priority of provisional application 62/160,545, filed on May 12, 2015.
Claims priority of provisional application 62/143,729, filed on Apr. 6, 2015.
Prior Publication US 2022/0195427 A1, Jun. 23, 2022
Int. Cl. C12N 15/11 (2006.01); C12N 9/22 (2006.01); C12N 9/96 (2006.01); C12N 15/90 (2006.01)
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.