Dual Supramolecular Nanoparticle Vectors Enable CRISPR/Cas9‐Mediated Knockin of Retinoschisin 1 Gene—A Potential Nonviral Therapeutic Solution for X‐Linked Juvenile Retinoschisis

Author:

Chou Shih‐Jie12,Yang Peng3ORCID,Ban Qian4,Yang Yi‐Ping56,Wang Mong‐Lien257,Chien Chian‐Shiu12,Chen Shih‐Jen12,Sun Na3,Zhu Yazhen3,Liu Hongtao8,Hui Wenqiao9,Lin Tai‐Chi12,Wang Fang10,Zhang Ryan Yue3,Nguyen Viet Q.12,Liu Wenfei11,Chen Mengxiang3,Jonas Steve J.12,Weiss Paul S.11ORCID,Tseng Hsian‐Rong3ORCID,Chiou Shih‐Hwa12

Affiliation:

1. Division of Basic ResearchDepartment of Medical Researchand Department of OphthalmologyTaipei Veterans General Hospital Taipei 112 Taiwan

2. Institute of PharmacologySchool of MedicineNational Yang‐Ming University Taipei 112 Taiwan

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

4. Center for Stem Cell and Translational MedicineSchool of Life SciencesAnhui University Hefei 230601 China

5. Department of Medical ResearchTaipei Veterans General Hospital Taipei 112 Taiwan

6. School of Medicine, and School of Pharmaceutical SciencesNational Yang‐Ming University Taipei 112 Taiwan

7. Institute of Food Safety and Health Risk AssessmentNational Yang Ming University Taipei 112 Taiwan

8. Shandong Provincial Qianfoshan Hospitalthe First Hospital Affiliated to Shandong First Medical University Jinan 250014 China

9. Institute of Animal Husbandry and Veterinary MedicineAnhui Academy of Agriculture Sciences Hefei 230031 China

10. State Key Laboratory of Molecular Engineering of PolymersDepartment of Macromolecular ScienceFudan University Shanghai 200433 China

11. Department of Chemistry and BiochemistryDepartment of BioengineeringDepartment of Materials Science and EngineeringCalifornia NanoSystems Institute (CNSI)University of California, Los Angeles Los Angeles CA 90095 USA

12. California NanoSystems Institute (CNSI)Department of PediatricsDavid Geffen School of MedicineEli & Edythe Broad Center of Regenerative Medicine and Stem Cell ResearchChildren's Discovery and Innovation InstituteUniversity of California, Los Angeles Los Angeles CA 90095 USA

Funder

National Institutes of Health

Taipei Veterans General Hospital

Ministry of Science and Technology, Taiwan

National Yang-Ming University

Ministry of Education

Publisher

Wiley

Subject

General Physics and Astronomy,General Engineering,Biochemistry, Genetics and Molecular Biology (miscellaneous),General Materials Science,General Chemical Engineering,Medicine (miscellaneous)

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