ArticleScience advances2020
Supramolecular nanosubstrate-mediated delivery system enables CRISPR-Cas9 knockin of hemoglobin beta gene for hemoglobinopathies.
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Article in Science advances, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.
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23citing papers in PubMed
2.3field-weighted citation impact, top 11% of its field
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23 citing papers in PubMed, 40 citations in OpenAlex.
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- pH-Responsive Polymer Nanoparticles for Efficient Delivery of Cas9 Ribonucleoprotein With or Without Donor DNA.Advanced materials (Deerfield Beach, Fla.) · 2022Article
- Nano-vectors for CRISPR/Cas9-Mediated Genome Editing.Nano today · 2022Article
- Research Progress on Gene Editing Based on Nano-Drug Delivery Vectors for Tumor Therapy.Frontiers in bioengineering and biotechnology · 2022Review
- CRISPR/Cas12a-Enhanced Loop-Mediated Isothermal Amplification for the Visual Detection ofFrontiers in bioengineering and biotechnology · 2022Article
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Corrections and comments
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Authors and funding
25 authors at 10 institutions in 3 countries.
Peng YangCenter for Stem Cell and Translational Medicine, School of Life Sciences, Anhui University, Hefei 230601, China.ORCID 0000-0003-0861-9687
Shih-Jie ChouDivision of Basic Research, Department of Medical Research, Taipei Veterans General Hospital, Taipei, Taiwan.ORCID 0000-0003-2610-8178
Jindian LiMolecular Imaging Center, Department of Radiology, Keck School of Medicine, University of Southern California, Los Angeles, CA 90033-9061, USA.
Wenqiao HuiInstitute of Animal Husbandry and Veterinary Medicine, Anhui Academy of Agriculture Sciences, Hefei 230031, China.
Wenfei LiuDepartment 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.ORCID 0000-0003-1338-7905
Na SunDepartment 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.ORCID 0000-0002-3282-4405
Ryan Y ZhangDepartment 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.ORCID 0000-0002-3122-4053
Yazhen ZhuDepartment 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.ORCID 0000-0002-2130-8085
Ming-Long TsaiDivision of Basic Research, Department of Medical Research, Taipei Veterans General Hospital, Taipei, Taiwan.ORCID 0000-0002-9957-923X
Henkie I LaiDivision of Basic Research, Department of Medical Research, Taipei Veterans General Hospital, Taipei, Taiwan.ORCID 0000-0001-8516-743X
Matthew SmalleyDepartment 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.ORCID 0000-0002-0509-6502
Xinyue ZhangDepartment 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.
Jiayuan ChenDepartment 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.ORCID 0000-0003-2586-8626
Zulema RomeroDepartment 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.ORCID 0000-0001-8305-8184
Dahai LiuSchool of Stomatology and Medicine, Foshan University, Foshan 528000, China.
Zunfu KeDepartment of Pathology, Institute of Precision Medicine, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou 510080, China.
Chang ZouClinical Medical Research Center, The First Affiliated Hospital of Southern University, Shenzhen People's Hospital, Shenzhen 518020, China.ORCID 0000-0002-2003-7834
Chin-Fa LeeDepartment 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.ORCID 0000-0003-0735-5691
Steven J JonasDepartment 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.ORCID 0000-0002-8111-0249
Qian BanCenter for Stem Cell and Translational Medicine, School of Life Sciences, Anhui University, Hefei 230601, China. hrtseng@mednet.ucla.edu shchiou@vghtpe.gov.tw chenkai@med.usc.edu dkohn1@mednet.ucla.edu psw@cnsi.ucla.edu bqashi@ahu.edu.cn.ORCID 0000-0002-3520-1721
Paul S WeissDepartment 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. hrtseng@mednet.ucla.edu shchiou@vghtpe.gov.tw chenkai@med.usc.edu dkohn1@mednet.ucla.edu psw@cnsi.ucla.edu bqashi@ahu.edu.cn.ORCID 0000-0001-5527-6248
Donald B KohnDepartment 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. hrtseng@mednet.ucla.edu shchiou@vghtpe.gov.tw chenkai@med.usc.edu dkohn1@mednet.ucla.edu psw@cnsi.ucla.edu bqashi@ahu.edu.cn.ORCID 0000-0003-1840-6087
Kai ChenMolecular Imaging Center, Department of Radiology, Keck School of Medicine, University of Southern California, Los Angeles, CA 90033-9061, USA. hrtseng@mednet.ucla.edu shchiou@vghtpe.gov.tw chenkai@med.usc.edu dkohn1@mednet.ucla.edu psw@cnsi.ucla.edu bqashi@ahu.edu.cn.ORCID 0000-0002-8647-1182
Shih-Hwa ChiouDepartment of Medical Research, Taipei Veterans General Hospital, Taipei, Taiwan. hrtseng@mednet.ucla.edu shchiou@vghtpe.gov.tw chenkai@med.usc.edu dkohn1@mednet.ucla.edu psw@cnsi.ucla.edu bqashi@ahu.edu.cn.ORCID 0000-0002-8651-5755
Hsian-Rong TsengDepartment 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. hrtseng@mednet.ucla.edu shchiou@vghtpe.gov.tw chenkai@med.usc.edu dkohn1@mednet.ucla.edu psw@cnsi.ucla.edu bqashi@ahu.edu.cn.ORCID 0000-0003-0942-5905
California NanoSystems Institute · USNational Yang Ming Chiao Tung University · TWBroad Center · USUniversity of Southern California · USAnhui Academy of Agricultural Sciences · CNAnhui University · CNFoshan University · CNNational Chung Hsing University · TWSouthern Medical University Shenzhen Hospital · CNSun Yat-sen University · CN
Funding
Nanoscience-Inspired Acoustofluidic Assembly Lines for Gene and Cellular TherapiesDP5OD028181 · OD · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI JONAS, STEVEN JOHN · 2019 to 2023
$1.9MSupramolecular Nanoparticle-Based PET Probes for Pretargeted Tumor ImagingR21EB016270 · NIBIB · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI TSENG, HSIAN-RONG · 2013 to 2014
$400kNIBIB NIH HHS R21 EB016270NIH HHS DP5 OD028181
Abstract
Leveraging the endogenous homology-directed repair (HDR) pathway, the CRISPR-Cas9 gene-editing system can be applied to knock in a therapeutic gene at a designated site in the genome, offering a general therapeutic solution for treating genetic diseases such as hemoglobinopathies. Here, a combined supramolecular nanoparticle (SMNP)/supramolecular nanosubstrate-mediated delivery (SNSMD) strategy is used to facilitate CRISPR-Cas9 knockin of the hemoglobin beta (HBB) gene into the adeno-associated virus integration site 1 (AAVS1) safe-harbor site of an engineered K562 3.21 cell line harboring the sickle cell disease mutation. Through stepwise treatments of the two SMNP vectors encapsulating a Cas9•single-guide RNA (sgRNA) complex and an HBB/green fluorescent protein (GFP)-encoding plasmid, CRISPR-Cas9 knockin was successfully achieved via HDR. Last, the HBB/GFP-knockin K562 3.21 cells were introduced into mice via intraperitoneal injection to show their in vivo proliferative potential. This proof-of-concept demonstration paves the way for general gene therapeutic solutions for treating hemoglobinopathies.
Indexed as
CRISPR-Cas SystemsHemoglobinopathiesAnimalsGene EditingGenetic VectorsHemoglobinsMiceHemoglobins
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