TY - JOUR
T1 - Progress and challenges towards CRISPR/Cas clinical translation
AU - Rosenblum, Daniel
AU - Gutkin, Anna
AU - Dammes, Niels
AU - Peer, Dan
PY - 2020/1
Y1 - 2020/1
N2 - CRISPR/Cas systems (clustered regularly interspaced short palindromic repeats) have emerged as powerful tools to manipulate the genome for both research and therapeutic purposes. However, the clinical use of this system is hindered by multiple challenges, such as the rate of off-target effects, editing efficiency, the efficacy of HDR, immunogenicity, as well as development of efficient and safe delivery vehicles that can carry these compounds. Tremendous efforts are being conducted to overcome these challenges, including the discovery and engineering of more precise and efficacious Cas nucleases. Moreover, in recent years multiple viral and non-viral delivery approaches have been explored for in vivo delivery of CRISPR components. Here, we summarize the available CRISPR/Cas toolbox for genome editing as well as the recently developed in vivo delivery vehicles for CRISPR/Cas system. Furthermore, we discuss the remaining challenges for successful clinical translation of this system and highlight the current clinical applications.
AB - CRISPR/Cas systems (clustered regularly interspaced short palindromic repeats) have emerged as powerful tools to manipulate the genome for both research and therapeutic purposes. However, the clinical use of this system is hindered by multiple challenges, such as the rate of off-target effects, editing efficiency, the efficacy of HDR, immunogenicity, as well as development of efficient and safe delivery vehicles that can carry these compounds. Tremendous efforts are being conducted to overcome these challenges, including the discovery and engineering of more precise and efficacious Cas nucleases. Moreover, in recent years multiple viral and non-viral delivery approaches have been explored for in vivo delivery of CRISPR components. Here, we summarize the available CRISPR/Cas toolbox for genome editing as well as the recently developed in vivo delivery vehicles for CRISPR/Cas system. Furthermore, we discuss the remaining challenges for successful clinical translation of this system and highlight the current clinical applications.
KW - CRISPR/Cas systems
KW - Cancer immunotherapy
KW - Challenges
KW - Clinical translation
KW - Delivery
KW - Gene therapy
KW - Non-viral vectors
KW - Viral vectors
UR - http://www.scopus.com/inward/record.url?scp=85088148133&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85088148133&partnerID=8YFLogxK
U2 - 10.1016/j.addr.2020.07.004
DO - 10.1016/j.addr.2020.07.004
M3 - Review article
C2 - 32659256
AN - SCOPUS:85088148133
VL - 154-155
SP - 176
EP - 186
JO - Advanced Drug Delivery Reviews
JF - Advanced Drug Delivery Reviews
SN - 0169-409X
ER -