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Nanoparticles-mediated CRISPR-Cas9 gene therapy in inherited retinal diseases: applications, challenges, and emerging opportunities.
Chien, Yueh; Hsiao, Yu-Jer; Chou, Shih-Jie; Lin, Ting-Yi; Yarmishyn, Aliaksandr A; Lai, Wei-Yi; Lee, Meng-Shiue; Lin, Yi-Ying; Lin, Tzu-Wei; Hwang, De-Kuang; Lin, Tai-Chi; Chiou, Shih-Hwa; Chen, Shih-Jen; Yang, Yi-Ping.
  • Chien Y; Department of Medical Research, Taipei Veterans General Hospital, 201, Section 2, Shi-Pai Road, Taipei, 11267, Taiwan, ROC.
  • Hsiao YJ; Institute of Pharmacology, National Yang-Ming Chiao Tung University, Taipei, 11221, Taiwan.
  • Chou SJ; School of Medicine, National Yang-Ming Chiao Tung University, Taipei, 11221, Taiwan.
  • Lin TY; Department of Medical Research, Taipei Veterans General Hospital, 201, Section 2, Shi-Pai Road, Taipei, 11267, Taiwan, ROC.
  • Yarmishyn AA; School of Medicine, National Yang-Ming Chiao Tung University, Taipei, 11221, Taiwan.
  • Lai WY; Department of Medical Research, Taipei Veterans General Hospital, 201, Section 2, Shi-Pai Road, Taipei, 11267, Taiwan, ROC.
  • Lee MS; Institute of Pharmacology, National Yang-Ming Chiao Tung University, Taipei, 11221, Taiwan.
  • Lin YY; Department of Medical Research, Taipei Veterans General Hospital, 201, Section 2, Shi-Pai Road, Taipei, 11267, Taiwan, ROC.
  • Lin TW; Department of Medicine, Kaohsiung Medical University, Kaohsiung, 80708, Taiwan.
  • Hwang DK; Doctoral Degree Program of Translational Medicine, National Yang Ming Chiao Tung University and Academia Sinica, Taipei, 11221, Taiwan.
  • Lin TC; Department of Medical Research, Taipei Veterans General Hospital, 201, Section 2, Shi-Pai Road, Taipei, 11267, Taiwan, ROC.
  • Chiou SH; Department of Medical Research, Taipei Veterans General Hospital, 201, Section 2, Shi-Pai Road, Taipei, 11267, Taiwan, ROC.
  • Chen SJ; Institute of Pharmacology, National Yang-Ming Chiao Tung University, Taipei, 11221, Taiwan.
  • Yang YP; School of Medicine, National Yang-Ming Chiao Tung University, Taipei, 11221, Taiwan.
J Nanobiotechnology ; 20(1): 511, 2022 Dec 03.
Article in English | MEDLINE | ID: covidwho-2254845
ABSTRACT
Inherited Retinal Diseases (IRDs) are considered one of the leading causes of blindness worldwide. However, the majority of them still lack a safe and effective treatment due to their complexity and genetic heterogeneity. Recently, gene therapy is gaining importance as an efficient strategy to address IRDs which were previously considered incurable. The development of the clustered regularly-interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9) system has strongly empowered the field of gene therapy. However, successful gene modifications rely on the efficient delivery of CRISPR-Cas9 components into the complex three-dimensional (3D) architecture of the human retinal tissue. Intriguing findings in the field of nanoparticles (NPs) meet all the criteria required for CRISPR-Cas9 delivery and have made a great contribution toward its therapeutic applications. In addition, exploiting induced pluripotent stem cell (iPSC) technology and in vitro 3D retinal organoids paved the way for prospective clinical trials of the CRISPR-Cas9 system in treating IRDs. This review highlights important advances in NP-based gene therapy, the CRISPR-Cas9 system, and iPSC-derived retinal organoids with a focus on IRDs. Collectively, these studies establish a multidisciplinary approach by integrating nanomedicine and stem cell technologies and demonstrate the utility of retina organoids in developing effective therapies for IRDs.
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Full text: Available Collection: International databases Database: MEDLINE Main subject: Retinal Diseases / Nanoparticles Type of study: Cohort study / Observational study / Prognostic study Limits: Humans Language: English Journal: J Nanobiotechnology Year: 2022 Document Type: Article

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Full text: Available Collection: International databases Database: MEDLINE Main subject: Retinal Diseases / Nanoparticles Type of study: Cohort study / Observational study / Prognostic study Limits: Humans Language: English Journal: J Nanobiotechnology Year: 2022 Document Type: Article