Supramolecular nanosubstrate–mediated delivery system enables CRISPR-Cas9 knockin of hemoglobin beta gene for hemoglobinopathies

Author:

Yang Peng12ORCID,Chou Shih-Jie34ORCID,Li Jindian5,Hui Wenqiao6,Liu Wenfei7ORCID,Sun Na2ORCID,Zhang Ryan Y.2ORCID,Zhu Yazhen2ORCID,Tsai Ming-Long34ORCID,Lai Henkie I.34ORCID,Smalley Matthew2ORCID,Zhang Xinyue2,Chen Jiayuan2ORCID,Romero Zulema8ORCID,Liu Dahai9,Ke Zunfu10,Zou Chang11ORCID,Lee Chin-Fa12ORCID,Jonas Steven J.13ORCID,Ban Qian1ORCID,Weiss Paul S.7ORCID,Kohn Donald B.8ORCID,Chen Kai5ORCID,Chiou Shih-Hwa14415ORCID,Tseng Hsian-Rong2ORCID

Affiliation:

1. Center for Stem Cell and Translational Medicine, School of Life Sciences, Anhui University, Hefei 230601, China.

2. Department of Molecular and Medical Pharmacology, Crump Institute for Molecular Imaging (CIMI), California NanoSystems Institute (CNSI), University of California, Los Angeles, Los Angeles, CA 90095, USA.

3. Division of Basic Research, Department of Medical Research, Taipei Veterans General Hospital, Taipei, Taiwan.

4. Institute of Pharmacology, School of Medicine, National Yang-Ming University, Taipei, Taiwan.

5. Molecular Imaging Center, Department of Radiology, Keck School of Medicine, University of Southern California, Los Angeles, CA 90033-9061, USA.

6. Institute of Animal Husbandry and Veterinary Medicine, Anhui Academy of Agriculture Sciences, Hefei 230031, China.

7. Department of Chemistry and Biochemistry, Department of Bioengineering, Department of Materials Science and Engineering, and California NanoSystems Institute (CNSI), University of California, Los Angeles, Los Angeles, CA 90095, USA.

8. Department of Microbiology, Immunology and Molecular Genetics, DGSOM, Division of Pediatric Hematology–Oncology, Department of Pediatrics, David Geffen School of Medicine, Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, Jonsson Comprehensive Cancer Center, University of California, Los Angeles, Los Angeles, CA 90095, USA.

9. School of Stomatology and Medicine, Foshan University, Foshan 528000, China.

10. Department of Pathology, Institute of Precision Medicine, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou 510080, China.

11. Clinical Medical Research Center, The First Affiliated Hospital of Southern University, Shenzhen People’s Hospital, Shenzhen 518020, China.

12. Department of Chemistry, i-Center for Advanced Science and Technology, Innovation and Development Center of Sustainable Agriculture, National Chung Hsing University (NCHU), 145 Xingda Road, South Dist., Taichung 402, Taiwan.

13. Department of Pediatrics, David Geffen School of Medicine, Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, Children’s Discovery and Innovation Institute, California NanoSystems Institute (CNSI), University of California, Los Angeles, Los Angeles, CA 90095, USA.

14. Department of Medical Research, Taipei Veterans General Hospital, Taipei, Taiwan.

15. Genomic Research Center, Academia Sinica, Taipei, Taiwan.

Abstract

CRISPR-Cas9 knockin of the HBB gene paves the way for a general therapeutic solution for treating hemoglobinopathies.

Funder

National Institutes of Health

Sorrento Therapeutics

Publisher

American Association for the Advancement of Science (AAAS)

Subject

Multidisciplinary

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