Abstract
Clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR-associated protein 9 (Cas9) technology has revolutionized therapeutic gene editing by providing researchers with a new method to study and cure diseases previously considered untreatable. While the full range and power of CRISPR technology for therapeutics is being elucidated through in vitro studies, translation to in vivo studies is slow. To date there is no totally effective delivery strategy to carry CRISPR components to the target site in vivo. The complexity of in vivo delivery is furthered by the number of potential delivery methods, the different forms in which CRISPR can be delivered as a therapeutic, and the disease target and tissue type in question. There are major challenges and limitations to delivery strategies, and it is imperative that future directions are guided by well-conducted studies that consider the full effect these variables have on the eventual outcome. In this review we will discuss the advances of the latest in vivo CRISPR/Cas9 delivery strategies and highlight the challenges yet to be overcome.
| Original language | English |
|---|---|
| Pages (from-to) | 304-311 |
| Number of pages | 8 |
| Journal | Biological and Pharmaceutical Bulletin |
| Volume | 42 |
| Issue number | 3 |
| DOIs | |
| Publication status | Published - Mar 2019 |
Cite this
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Advances in CRISPR/Cas9 Technology for in Vivo Translation. / Cicek, Yagiz Anil; Luther, David C.; Kretzmann, Jessica A.; Rotello, Vincent M.
In: Biological and Pharmaceutical Bulletin, Vol. 42, No. 3, 03.2019, p. 304-311.Research output: Contribution to journal › Review article
TY - JOUR
T1 - Advances in CRISPR/Cas9 Technology for in Vivo Translation
AU - Cicek, Yagiz Anil
AU - Luther, David C.
AU - Kretzmann, Jessica A.
AU - Rotello, Vincent M.
PY - 2019/3
Y1 - 2019/3
N2 - Clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR-associated protein 9 (Cas9) technology has revolutionized therapeutic gene editing by providing researchers with a new method to study and cure diseases previously considered untreatable. While the full range and power of CRISPR technology for therapeutics is being elucidated through in vitro studies, translation to in vivo studies is slow. To date there is no totally effective delivery strategy to carry CRISPR components to the target site in vivo. The complexity of in vivo delivery is furthered by the number of potential delivery methods, the different forms in which CRISPR can be delivered as a therapeutic, and the disease target and tissue type in question. There are major challenges and limitations to delivery strategies, and it is imperative that future directions are guided by well-conducted studies that consider the full effect these variables have on the eventual outcome. In this review we will discuss the advances of the latest in vivo CRISPR/Cas9 delivery strategies and highlight the challenges yet to be overcome.
AB - Clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR-associated protein 9 (Cas9) technology has revolutionized therapeutic gene editing by providing researchers with a new method to study and cure diseases previously considered untreatable. While the full range and power of CRISPR technology for therapeutics is being elucidated through in vitro studies, translation to in vivo studies is slow. To date there is no totally effective delivery strategy to carry CRISPR components to the target site in vivo. The complexity of in vivo delivery is furthered by the number of potential delivery methods, the different forms in which CRISPR can be delivered as a therapeutic, and the disease target and tissue type in question. There are major challenges and limitations to delivery strategies, and it is imperative that future directions are guided by well-conducted studies that consider the full effect these variables have on the eventual outcome. In this review we will discuss the advances of the latest in vivo CRISPR/Cas9 delivery strategies and highlight the challenges yet to be overcome.
KW - clustered regularly interspaced short palindromic repeat
KW - genomics
KW - therapeutics
KW - in vivo
KW - HOMOLOGY-DIRECTED-REPAIR
KW - MOUSE MODEL
KW - MESSENGER-RNA
KW - GENE DELIVERY
KW - ADENOASSOCIATED VIRUS
KW - NANOPARTICLE DELIVERY
KW - NONVIRAL VECTORS
KW - GENOME
KW - ADENOVIRUS
KW - CAS9
U2 - 10.1248/bpb.b18-00811
DO - 10.1248/bpb.b18-00811
M3 - Review article
VL - 42
SP - 304
EP - 311
JO - Biological and Pharmaceutical Bulletin
JF - Biological and Pharmaceutical Bulletin
SN - 0918-6158
IS - 3
ER -