Combined liver–cytokine humanization comes to the rescue of circulating human red blood cells-Reference-Cited by-同舟云学术

Combined liver–cytokine humanization comes to the rescue of circulating human red blood cells

Published:2021-03-05 Issue:6533 Volume:371 Page:1019-1025
ISSN:0036-8075
Container-title:Science
language:en
Short-container-title:Science

Author:

Song Yuanbin¹²³^ORCID,Shan Liang⁴⁵^ORCID,Gbyli Rana¹²,Liu Wei¹²,Strowig Till⁴⁶^ORCID,Patel Amisha¹²,Fu Xiaoying¹²⁷^ORCID,Wang Xiaman¹²⁸^ORCID,Xu Mina L.⁹^ORCID,Gao Yimeng¹²^ORCID,Qin Ashley¹²^ORCID,Bruscia Emanuela M.¹⁰^ORCID,Tebaldi Toma¹²¹¹^ORCID,Biancon Giulia¹²^ORCID,Mamillapalli Padmavathi¹²,Urbonas David⁴^ORCID,Eynon Elizabeth⁴^ORCID,Gonzalez David G.¹²^ORCID,Chen Jie⁴^ORCID,Krause Diane S.²⁹¹³,Alderman Jonathan⁴,Halene Stephanie¹²^ORCID,Flavell Richard A.⁴¹⁴^ORCID

Affiliation:

1. Section of Hematology, Department of Internal Medicine, Yale Cancer Center, and Yale Center for RNA Science and Medicine, Yale University School of Medicine, New Haven, CT, USA.

2. Yale Stem Cell Center, Yale University School of Medicine, New Haven, CT, USA.

3. Department of Hematologic Oncology, Sun Yat-sen University Cancer Center, State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, China.

4. Department of Immunobiology, Yale University School of Medicine, New Haven, CT, USA.

5. Department of Medicine, Pathology and Immunology, Center for Human Immunology and Immunotherapy Programs, Washington University School of Medicine, St. Louis, MO, USA.

6. Helmholtz Centre for Infection Research, Braunschweig, Germany.

7. Department of Laboratory Medicine, Shenzhen Children’s Hospital, Shenzhen, People’s Republic of China.

8. Department of Hematology, the Second Affiliated Hospital of Xi’an Jiaotong University, Xi’an, People’s Republic of China.

9. Department of Pathology, Yale University School of Medicine, New Haven, CT, USA.

10. Department of Pediatrics, Yale University School of Medicine, New Haven, CT, USA.

11. Department of Cellular, Computational and Integrative Biology (CIBIO), University of Trento, Trento, Italy.

12. Department of Genetics, Yale University School of Medicine, New Haven, CT, USA.

13. Department of Laboratory Medicine, Yale School of Medicine, New Haven, CT, USA.

14. Howard Hughes Medical Institute, Yale University, New Haven, CT, USA.

Abstract

A red-letter day for RBC research The study of primary human red blood cell (huRBC) disorders such as sickle cell disease (SCD) and infectious diseases such as malaria has been hampered by a lack of in vivo models of human erythropoiesis. Song et al. transferred human fetal liver cells into MISTRG mice, which are immunodeficient and are genetically engineered with several human genes involved in hematopoiesis. This approach was unsuccessful because mature huRBCs are rapidly destroyed in the mouse liver. They then used CRISPR-Cas9 to mutate these mice into a fumarylacetoacetate hydrolase–deficient strain, allowing them to replace the mouse liver with engrafted human hepatocytes. These mice exhibited enhanced human erythropoiesis and circulating huRBC survival and could recapitulate SCD pathology when reconstituted with SCD-derived HSCs. Science , this issue p. 1019

Funder

Howard Hughes Medical Institute

Bill and Melinda Gates Foundation

NIDDK

NIH/NIDDK

Young Scientists Fund of the National Natural Science Foundation of China

The Frederick A. Deluca Foundation

James Hudson Brown - Alexander Brown Coxe Postdoctoral Fellowships

Publisher

American Association for the Advancement of Science (AAAS)

Subject

Multidisciplinary