Projects per year
Abstract
Introduction: Gene therapy is becoming increasingly common in clinical practice, giving hope for the correction of a wide range of human diseases and defects. The CRISPR/Cas9 system, consisting of the Cas9 nuclease and single-guide RNA (sgRNA), has revolutionized the field of gene editing. However, efficiently delivering the CRISPR-Cas9 to the target organ or cell remains a significant challenge. In recent years, with rapid advances in nanoscience, materials science, and medicine, researchers have developed various technologies that can deliver CRISPR-Cas9 in different forms for in vitro and in vivo gene editing. Here, we review the development of the CRISPR-Cas9 and describe the delivery forms and the vectors that have emerged in CRISPR-Cas9 delivery, summarizing the key barriers and the promising strategies that vectors currently face in delivering the CRISPR-Cas9. Areas covered: With the rapid development of CRISPR-Cas9, delivery methods are becoming increasingly important in the in vivo delivery of CRISPR-Cas9. Expert opinion: CRISPR-Cas9 is becoming increasingly common in clinical trials. However, the complex nuclease and protease environment is a tremendous challenge for in vivo clinical applications. Therefore, the development of delivery methods is highly likely to take the application of CRISPR-Cas9 technology to another level.
Original language | English |
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Pages (from-to) | 913-926 |
Number of pages | 14 |
Journal | Expert Opinion on Drug Delivery |
Volume | 19 |
Issue number | 8 |
DOIs | |
Publication status | Published - 17 Jul 2022 |
MoE publication type | A2 Review article in a scientific journal |
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Åbo Akademi Functional Printing Center
Toivakka, M. (PI), Rosenholm, J. (PI), Anttu, N. (PI), Bobacka, J. (PI), Huynh, T. P. (PI), Peltonen, J. (PI), Wang, X. (PI), Wilen, C.-E. (PI), Xu, C. (PI), Zhang, H. (PI) & Österbacka, R. (PI)
Faculty of Science and EngineeringFacility/equipment: Facility
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FCFH: Finland-China Network in Food and Health Sciences
Rosenholm, J. (Principal Investigator), Xu, C. (Principal Investigator) & Zhang, H. (Principal Investigator)
Ministry of Education and Culture
01/01/21 → 31/12/24
Project: Ministry / Government Agency
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Targeted delivery of CRISPR/Cas9 for advanced liver cancer therapy through c-Myc knockout
Zhang, H. (Principal Investigator)
01/09/19 → 31/08/24
Project: Research Council of Finland/Other Research Councils