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.
SormenjälkiSukella tutkimusaiheisiin 'Progress of delivery methods for CRISPR-Cas9'. Ne muodostavat yhdessä ainutlaatuisen sormenjäljen.
Martti Toivakka (PI), Jessica Rosenholm (PI), Nicklas Anttu (PI), Johan Bobacka (PI), Tan Phat Huynh (PI), Jouko Peltonen (PI), Xiaoju Wang (PI), Carl-Eric Wilen (PI), Chunlin Xu (PI), Hongbo Zhang (PI) & Ronald Österbacka (PI)Faculty of Science and Engineering
- 2 Aktiivinen
01/01/21 → 31/12/24
Projekti: Ministry / Government Agency
01/09/19 → 31/08/24
Projekti: Academy of Finland/Other Research Councils