Title:
TARGETED INTEGRATION AT ALPHA-GLOBIN LOCUS IN HUMAN HEMATOPOIETIC STEM AND PROGENITOR CELLS
Document Type and Number:
WIPO Patent Application WO/2023/224992
Kind Code:
A3
Abstract:
The present disclosure provides methods and compositions for genetically modifying hematopoietic stem and progenitor cells (HSPCs), in particular by replacing the HBA1 or HBA2 locus in the HSPCs with a transgene encoding a therapeutic protein.
Inventors:
PORTEUS MATTHEW H (US)
CROMER MICHAEL KYLE (US)
HAMPTON JESSICA P (US)
AMORIN ALVARO (US)
CROMER MICHAEL KYLE (US)
HAMPTON JESSICA P (US)
AMORIN ALVARO (US)
Application Number:
PCT/US2023/022379
Publication Date:
March 14, 2024
Filing Date:
May 16, 2023
Export Citation:
Assignee:
UNIV LELAND STANFORD JUNIOR (US)
International Classes:
A61K35/14; A61K38/36; C12N5/078; C12N5/0797; C12N15/63; C12N15/90; A61K35/545
Domestic Patent References:
| WO2021097350A1 | 2021-05-20 | |||
| WO2021160758A2 | 2021-08-19 |
Foreign References:
| US20210230570A1 | 2021-07-29 | |||
| US20150283267A1 | 2015-10-08 |
Other References:
JIA YI, WARIN RENAUD, YU XIAOBING, EPSTEIN REED, NOGUCHI CONSTANCE TOM: "Erythropoietin signaling promotes transplanted progenitor cell survival", THE FASEB JOURNAL, vol. 23, no. 9, 1 September 2009 (2009-09-01), US, pages 3089 - 3099, XP093149082, ISSN: 0892-6638, DOI: 10.1096/fj.09-130237
Attorney, Agent or Firm:
YAN, Lin et al. (US)
Download PDF:
Previous Patent: DOCK ASSEMBLY FOR UNMANNED AERIAL VEHICLES
Next Patent: RAPID DETECTION TESTS AND METHODS OF FORMING THE SAME
Next Patent: RAPID DETECTION TESTS AND METHODS OF FORMING THE SAME