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Supramolecular nanosubstrate-mediated delivery system enables CRISPR-Cas9 knockin of hemoglobin beta gene for hemoglobinopathies.
Yang, Peng; Chou, Shih-Jie; Li, Jindian; Hui, Wenqiao; Liu, Wenfei; Sun, Na; Zhang, Ryan Y; Zhu, Yazhen; Tsai, Ming-Long; Lai, Henkie I; Smalley, Matthew; Zhang, Xinyue; Chen, Jiayuan; Romero, Zulema; Liu, Dahai; Ke, Zunfu; Zou, Chang; Lee, Chin-Fa; Jonas, Steven J; Ban, Qian; Weiss, Paul S; Kohn, Donald B; Chen, Kai; Chiou, Shih-Hwa; Tseng, Hsian-Rong.
Afiliación
  • Yang P; Center for Stem Cell and Translational Medicine, School of Life Sciences, Anhui University, Hefei 230601, China.
  • Chou SJ; 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.
  • Li J; Division of Basic Research, Department of Medical Research, Taipei Veterans General Hospital, Taipei, Taiwan.
  • Hui W; Institute of Pharmacology, School of Medicine, National Yang-Ming University, Taipei, Taiwan.
  • Liu W; Molecular Imaging Center, Department of Radiology, Keck School of Medicine, University of Southern California, Los Angeles, CA 90033-9061, USA.
  • Sun N; Institute of Animal Husbandry and Veterinary Medicine, Anhui Academy of Agriculture Sciences, Hefei 230031, China.
  • Zhang RY; 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.
  • Zhu Y; 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.
  • Tsai ML; 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.
  • Lai HI; 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.
  • Smalley M; Division of Basic Research, Department of Medical Research, Taipei Veterans General Hospital, Taipei, Taiwan.
  • Zhang X; Institute of Pharmacology, School of Medicine, National Yang-Ming University, Taipei, Taiwan.
  • Chen J; Division of Basic Research, Department of Medical Research, Taipei Veterans General Hospital, Taipei, Taiwan.
  • Romero Z; Institute of Pharmacology, School of Medicine, National Yang-Ming University, Taipei, Taiwan.
  • Liu D; 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.
  • Ke Z; 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.
  • Zou C; 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.
  • Lee CF; 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
  • Jonas SJ; School of Stomatology and Medicine, Foshan University, Foshan 528000, China.
  • Ban Q; Department of Pathology, Institute of Precision Medicine, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou 510080, China.
  • Weiss PS; Clinical Medical Research Center, The First Affiliated Hospital of Southern University, Shenzhen People's Hospital, Shenzhen 518020, China.
  • Kohn DB; 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.
  • Chen K; 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.
  • Chiou SH; Center 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.
  • Tseng HR; 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. hrtseng@mednet.ucla.edu shchiou@vghtpe.gov.tw chenkai@med.usc.edu d
Sci Adv ; 6(43)2020 10.
Article en En | MEDLINE | ID: mdl-33097539
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Sistemas CRISPR-Cas / Hemoglobinopatías Límite: Animals Idioma: En Revista: Sci Adv Año: 2020 Tipo del documento: Article País de afiliación: China
Texto completo
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PubMed Links
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Sistemas CRISPR-Cas / Hemoglobinopatías Límite: Animals Idioma: En Revista: Sci Adv Año: 2020 Tipo del documento: Article País de afiliación: China