Title:
DIFFERENTIAL PROLIFERATION OF HUMAN HEMATOPOIETIC STEM AND PROGENITOR CELLS USING TRUNCATED ERYTHROPOIETIN RECEPTORS
Document Type and Number:
WIPO Patent Application WO/2023/064798
Kind Code:
A3
Abstract:
Edited cell chimerism is currently one of the greatest bottlenecks to clinical efficacy of gene therapies for the hemoglobinopathies. For example, it is difficult to go from low hematopoietic stem cell (HSC) edited ceil chimerism in the bone marrow to high edited red blood cell (RBC) chimerism in the bloodstream. The present disclosure provides methods and compositions for genetically modifying hematopoietic stem and progenitor cells (HSPCs), in particular by creating HSPCs that express truncated forms of the EPO.receptor (tEPOR).
Inventors:
PORTEUS MATTHEW H (US)
CROMER MICHAEL KYLE (US)
CAMARENA JOAB (US)
CROMER MICHAEL KYLE (US)
CAMARENA JOAB (US)
Application Number:
PCT/US2022/077951
Publication Date:
November 16, 2023
Filing Date:
October 12, 2022
Export Citation:
Assignee:
UNIV LELAND STANFORD JUNIOR (US)
International Classes:
C12N5/00; A61K38/18; A61K48/00; A61P7/00; C07K14/71; C07K14/805; C12N5/10; C12N9/22
Domestic Patent References:
| WO2021097350A1 | 2021-05-20 | |||
| WO2021154791A1 | 2021-08-05 | |||
| WO2018011805A2 | 2018-01-18 |
Foreign References:
| US20180201952A1 | 2018-07-19 | |||
| US20210171983A1 | 2021-06-10 | |||
| US20110077283A1 | 2011-03-31 | |||
| US20040197873A1 | 2004-10-07 | |||
| US20210261985A1 | 2021-08-26 |
Other References:
NIU JIAYAO: "One-Time Treatment of Ketamine Alleviate Fear Memory and Spatial Cognition Deficits in PTSD Rat Models via BDNF Signaling and Post-Synaptic Density 95 of Hippocampus and Amygdala", INTERNATIONAL JOURNAL OF PSYCHOPHYSIOLOGY, ELSEVIER, AMSTERDAM, NL, vol. 168, 7 September 2021 (2021-09-07), NL , XP086772096, ISSN: 0167-8760, DOI: 10.1016/j.ijpsycho.2021.07.080
YOON DONGHOON; PRCHAL JOSEF: "Truncated Human Erythropoietin Receptor Causes Fetal Polycythemia and Is Associated with a Prolonged Primitive Erythropoiesis and Hyperactive Definitive Erythropoiesis.", BLOOD, AMERICAN SOCIETY OF HEMATOLOGY, US, vol. 110, no. 11, 16 November 2007 (2007-11-16), US , pages 3664, XP086644291, ISSN: 0006-4971, DOI: 10.1182/blood.V110.11.3664.3664
MEGAN BASILA, MELISSA KELLEY, ANJA VAN BRABANT SMITH, STEFAN MAAS: "Minimal 2'-O-methyl phosphorothioate linkage modification pattern of synthetic guide RNAs for increased stability and efficient CRISPR-Cas9 gene editing avoiding cellular toxicity", PLOS ONE, PUBLIC LIBRARY OF SCIENCE, vol. 12, no. 11, 27 November 2017 (2017-11-27), pages e0188593, XP055569679, DOI: 10.1371/journal.pone.0188593
YOON DONGHOON; PRCHAL JOSEF: "Truncated Human Erythropoietin Receptor Causes Fetal Polycythemia and Is Associated with a Prolonged Primitive Erythropoiesis and Hyperactive Definitive Erythropoiesis.", BLOOD, AMERICAN SOCIETY OF HEMATOLOGY, US, vol. 110, no. 11, 16 November 2007 (2007-11-16), US , pages 3664, XP086644291, ISSN: 0006-4971, DOI: 10.1182/blood.V110.11.3664.3664
MEGAN BASILA, MELISSA KELLEY, ANJA VAN BRABANT SMITH, STEFAN MAAS: "Minimal 2'-O-methyl phosphorothioate linkage modification pattern of synthetic guide RNAs for increased stability and efficient CRISPR-Cas9 gene editing avoiding cellular toxicity", PLOS ONE, PUBLIC LIBRARY OF SCIENCE, vol. 12, no. 11, 27 November 2017 (2017-11-27), pages e0188593, XP055569679, DOI: 10.1371/journal.pone.0188593
Attorney, Agent or Firm:
HAO, Joe C. et al. (US)
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