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journal article·2020-05Open access

Gene Therapy: Dual Supramolecular Nanoparticle Vectors Enable CRISPR/Cas9‐Mediated Knockin of Retinoschisin 1 Gene—A Potential Nonviral Therapeutic Solution for X‐Linked Juvenile Retinoschisis (Adv. Sci. 10/2020)

Shih‐Jie Chou, Peng Yang, Qian Ban, Yi‐Ping Yang, Mong‐Lien Wang, Chian‐Shiu Chien, Shih‐Jen Chen, Na Sun, Yazhen Zhu, Hongtao Liu, Wenqiao Hui, Tai‐Chi Lin, Fang Wang, Ryan Yue Zhang, Viet Q. Nguyen, Wenfei Liu, Mengxiang Chen, Steve J. Jonas, Paul S. Weiss, Hsian‐Rong Tseng, Shih‐Hwa Chiou

Advanced Science· Volume 7· Issue 10
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Source abstract

Abstract

In article number 1903432, Shih-Hwa Chiou, Hsian-Rong Tseng, Paul S. Weiss, and co-workers introduce an in vivo CRISPR/Cas9-mediated knockin approach using two supramolecular nanoparticle (SMNP) vectors. By intravitreally injecting the two SMNP vectors into the mouse eyes, the RS1/GFP gene is knocked into the Rosa26 site in mice retinas, offering a revolutionarily curative therapeutic solution for X-linked juvenile retinoschisis.
Source: openalex_inverted_index · Quality: baseline

Research context

Nanomaterials

OpenAlex topics: Retinal Development and Disorders · Advanced biosensing and bioanalysis techniques · Retinopathy of Prematurity Studies

SciVX discovery tags are terminology-based. OpenAlex topics are source-provided classifications.

Affiliations

Division of Basic Research Department of Medical Research and Department of Ophthalmology Taipei Veterans General Hospital Taipei 112 Taiwan

Institute of Pharmacology School of Medicine National Yang‐Ming University Taipei 112 Taiwan

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

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

Department of Medical Research Taipei Veterans General Hospital Taipei 112 Taiwan

School of Medicine, and School of Pharmaceutical Sciences National Yang‐Ming University Taipei 112 Taiwan

Institute of Food Safety and Health Risk Assessment National Yang Ming University Taipei 112 Taiwan

Shandong Provincial Qianfoshan Hospital the First Hospital Affiliated to Shandong First Medical University Jinan 250014 China

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

State Key Laboratory of Molecular Engineering of Polymers Department of Macromolecular Science Fudan University Shanghai 200433 China

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

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

References

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