Match!

CRISPR-Cas9 gene editing: Delivery aspects and therapeutic potential.

Published on Dec 1, 2016in Journal of Controlled Release7.901
· DOI :10.1016/j.jconrel.2016.08.002
Erik Oude Blenke7
Estimated H-index: 7
(UU: Utrecht University),
Martijn J. W. Evers3
Estimated H-index: 3
(UU: Utrecht University)
+ 1 AuthorsJ. van der Oost70
Estimated H-index: 70
(WUR: Wageningen University and Research Centre)
Abstract
The CRISPR-Cas9 gene editing system has taken the biomedical science field by storm, initiating rumors about future Nobel Prizes and heating up a fierce patent war, but also making significant scientific impact. The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR), together with CRISPR-associated proteins (Cas) are a part of the prokaryotic adaptive immune system and have successfully been repurposed for genome editing in mammalian cells. The CRISPR-Cas9 system has been used to correct genetic mutations and for replacing entire genes, opening up a world of possibilities for the treatment of genetic diseases. In addition, recently some new CRISPR-Cas systems have been discovered with interesting mechanistic variations. Despite these promising developments, many challenges have to be overcome before the system can be applied therapeutically in human patients and enabling delivery technology is one of the key challenges. Furthermore, the relatively high off-target effect of the system in its current form prevents it from being safely applied directly in the human body. In this review, the transformation of the CRISPR-Cas gene editing systems into a therapeutic modality will be discussed and the currently most realistic in vivo applications will be highlighted.
  • References (97)
  • Citations (23)
📖 Papers frequently viewed together
201241.06Science
5,693 Citations
42 Citations
8 Citations
78% of Scinapse members use related papers. After signing in, all features are FREE.
References97
Newest
#1Bernd Zetsche (MIT: Massachusetts Institute of Technology)H-Index: 6
#2Matthias Heidenreich (MIT: Massachusetts Institute of Technology)H-Index: 1
Last. Feng Zhang (MIT: Massachusetts Institute of Technology)H-Index: 118
view all 15 authors...
Multiplexed genome editing is simplified by harnessing the ability of Cpf1 to process its own pre-crRNA.
242 CitationsSource
#1Van Trung Chu (MDC: Max Delbrück Center for Molecular Medicine)H-Index: 12
#2Timm Weber (MDC: Max Delbrück Center for Molecular Medicine)H-Index: 4
Last. Ralf Kühn (MDC: Max Delbrück Center for Molecular Medicine)H-Index: 51
view all 9 authors...
The CRISPR/Cas9 system is increasingly used for gene inactivation in mouse zygotes, but homology-directed mutagenesis and use of inbred embryos are less established. In particular, Rosa26 knock-in alleles for the insertion of transgenes in a genomic ‘safe harbor’ site, have not been produced. Here we applied CRISPR/Cas9 for the knock-in of 8–11 kb inserts into Rosa26 of C57BL/6 zygotes. We found that 10–20 % of live pups derived from microinjected zygotes were founder mutants, without apparent o...
59 CitationsSource
Both RNA interference (RNAi) and clustered regularly-interspaced short palindromic repeats (CRISPR) technologies allow for the sequence-specific inhibition of gene function and therefore have the potential to be used as therapeutic modalities. By judging the current public and scientific journal interest, it would seem that CRISPR, by enabling clean, durable knockouts, will dominate therapeutic gene inhibition, also at the expense of RNAi. This review aims to look behind prevailing sentiments an...
7 CitationsSource
#1Prarthana Mohanraju (WUR: Wageningen University and Research Centre)H-Index: 7
#2Kira S. Makarova (NIH: National Institutes of Health)H-Index: 80
Last. J. van der Oost (WUR: Wageningen University and Research Centre)H-Index: 70
view all 6 authors...
BACKGROUND Prokaryotes have evolved multiple systems to combat invaders such as viruses and plasmids. Examples of such defense systems include receptor masking, restriction-modification (R-M) systems, DNA interference (Argonaute), bacteriophage exclusion (BREX or PGL), and abortive infection, all of which act in an innate, nonspecific manner. In addition, prokaryotes have evolved adaptive, heritable immune systems: clustered regularly interspaced palindromic repeats (CRISPR) and the CRISPR-assoc...
236 CitationsSource
#1Ioannis Mougiakos (WUR: Wageningen University and Research Centre)H-Index: 4
#2Elleke Fenna Bosma (WUR: Wageningen University and Research Centre)H-Index: 10
Last. J. van der Oost (WUR: Wageningen University and Research Centre)H-Index: 70
view all 5 authors...
The increasing demand for environmentally friendly production processes of green chemicals and fuels has stimulated research in microbial metabolic engineering. CRISPR-Cas-based tools for genome editing and expression control have enabled fast, easy, and accurate strain development for established production platform organisms, such as Escherichia coli and Saccharomyces cerevisiae. However, the growing interest in alternative production hosts, for which genome editing options are generally limit...
59 CitationsSource
44 CitationsSource
9 CitationsSource
#1M. Carrie Miceli (UCLA: University of California, Los Angeles)H-Index: 21
#2Stanley F. Nelson (UCLA: University of California, Los Angeles)H-Index: 85
9 CitationsSource
#1Ines Fonfara (MPG: Max Planck Society)H-Index: 7
#2Hagen Richter (MPG: Max Planck Society)H-Index: 10
Last. Emmanuelle Charpentier (MPG: Max Planck Society)H-Index: 30
view all 5 authors...
CRISPR-Cas systems that provide defence against mobile genetic elements in bacteria and archaea have evolved a variety of mechanisms to target and cleave RNA or DNA(1). The well-studied types I, II ...
280 CitationsSource
#1Rafal Kaminski (TU: Temple University)H-Index: 12
#2Yilan Chen (TU: Temple University)H-Index: 2
Last. Kamel Khalili (TU: Temple University)H-Index: 61
view all 9 authors...
We employed an RNA-guided CRISPR/Cas9 DNA editing system to precisely remove the entire HIV-1 genome spanning between 5′ and 3′ LTRs of integrated HIV-1 proviral DNA copies from latently infected human CD4+ T-cells. Comprehensive assessment of whole-genome sequencing of HIV-1 eradicated cells ruled out any off-target effects by our CRISPR/Cas9 technology that might compromise the integrity of the host genome and further showed no effect on several cell health indices including viability, cell cy...
134 CitationsSource
Cited By23
Newest
#1Yan Gong (MUC: Minzu University of China)
#2Siyu Tian (MUC: Minzu University of China)
Last. Shubiao Zhang (MUC: Minzu University of China)H-Index: 20
view all 4 authors...
The clustered regularly interspaced short palindromic repeats (CRISPR)/associated protein 9 (CRISPR/Cas9) system is the most widely used approach for gene editing. Due to the advantages of simple operation, high efficiency and high specificity, it has been used in various fields. In this review, we introduce the latest researches in the delivery technology of CRISPR/Cas9 based on lipids, polymers and polymer-modified inorganic nanoparticles, which are synthesized through chemical methods. Many s...
Source
#1Shahin Aghamiri (Shahid Beheshti University of Medical Sciences and Health Services)H-Index: 2
#2Sam Talaei (Shahid Beheshti University of Medical Sciences and Health Services)H-Index: 3
Last. Vahid Jajarmi (Shahid Beheshti University of Medical Sciences and Health Services)H-Index: 2
view all 7 authors...
Abstract Clustered regularly interspaced short palindromic repeat-CRISPR-associated protein (CRISPR-Cas) genome-editing systems, prokaryotic adaptive immune system, have been exploited and prepared a robust programmable method for efficient and accurate genome editing and gene targeting. This revolutionary technique can be used for engineered cell therapies, in vivo gene therapy, animal modeling, and cancer diagnosis and treatment. Recently, safe and efficient delivery is one of the greatest cha...
1 CitationsSource
#1Jun Ren (CAU: Chung-Ang University)H-Index: 2
#2Jingyu Lee (CAU: Chung-Ang University)H-Index: 1
Last. Dokyun Na (CAU: Chung-Ang University)H-Index: 14
view all 3 authors...
Genome-scale engineering is a crucial methodology to rationally regulate microbiological system operations, leading to expected biological behaviors or enhanced bioproduct yields. Over the past decade, innovative genome modification technologies have been developed for effectively regulating and manipulating genes at the genome level. Here, we discuss the current genome-scale engineering technologies used for microbial engineering. Recently developed strategies, such as clustered regularly inter...
1 CitationsSource
#1Trung Thanh Thach (SKKU: Sungkyunkwan University)H-Index: 4
#2Do Hyun Bae (SKKU: Sungkyunkwan University)
Last. Yong Ho Kim (SKKU: Sungkyunkwan University)H-Index: 9
view all 13 authors...
: A transient cytosolic delivery system for accurate Cas9 ribonucleoprotein is a key factor for target specificity of the CRIPSR/Cas9 toolkit. Owing to the large size of the Cas9 protein and a long negative strand RNA, the development of the delivery system is still a major challenge. Here, a size-controlled lipopeptide-based nanosome system is reported, derived from the blood-brain barrier-permeable dNP2 peptide which is capable of delivering a hyperaccurate Cas9 ribonucleoprotein complex (Hypa...
Source
#1Weiyu Zhao (OSU: Ohio State University)H-Index: 7
#2Xucheng Hou (OSU: Ohio State University)H-Index: 3
Last. Yizhou DongH-Index: 24
view all 4 authors...
Abstract Genetic and rare diseases (GARDs) affect more than 350 million patients worldwide and remain a significant challenge in the clinic. Hence, continuous efforts have been made to bridge the significant gap between the supply and demand of effective treatments for GARDs. Recent decades have witnessed the impressive progress in the fight against GARDs, with an improved understanding of the genetic origins of rare diseases and the rapid development in gene therapy providing a new avenue for G...
Source
#1Maria João Almeida (UC: University of Coimbra)H-Index: 2
Last. Matos A
view all 2 authors...
: Acquired Immunodeficiency Syndrome (AIDS), caused by the Human Immunodeficiency Virus (HIV), is a life-threatening disorder that persists worldwide as a severe health problem. Since it was linked with the HIV attachment process, the Chemokine receptor, CCR5, has been at the development leading edge of several gene-based therapies. Given the shortcomings of the current antiretroviral treatment procedure and the non-availability of a licensed vaccine, the aptitude to modify complex genomes with ...
2 CitationsSource
#1Niki D.J. Ubags (UNIL: University of Lausanne)H-Index: 10
#2Manuela Platé (UCL: University College London)H-Index: 10
Last. Sabine Bartel (UMCG: University Medical Center Groningen)H-Index: 1
view all 4 authors...
The #ERSCongress is the largest respiratory conference worldwide. This article points out why @ERSTalk Early Career Members can especially benefit from attending this huge event and gives upcoming fellowship deadlines. http://ow.ly/3GQ530o8Ezh.
Source
#1Marko Z. Nikolić (UCL: University College London)H-Index: 1
Last. Sabine BartelH-Index: 1
view all 9 authors...
The European Respiratory Society (ERS) International Congress is the largest respiratory congress and brings together leading experts in all fields of respiratory medicine and research. ERS Assembly 3 shapes the basic and translational science aspects of this congress, aiming to combine cutting-edge novel developments in basic research with novel clinical findings. In this article, we summarise a selection of the scientific highlights from the perspective of the three groups within Assembly 3. I...
Source
#1Jia Hui LeeH-Index: 4
#2Jiang-Hui Wang (University of Melbourne)H-Index: 5
Last. Guei-Sheung LiuH-Index: 20
view all 7 authors...
Many clinical trials using gene therapy have shown significant therapeutic benefits and exceptional safety records. Increasing evidence is verifying the long sought-after promise that gene therapy will genetically 'cure' some severely disabling diseases. In particular, the first gene therapy bioproduct for RPE65-associated Leber's congenital amaurosis, which was approved by the US Food and Drug Administration in 2017, has provided tremendous encouragement to the field of gene therapy. Recent dev...
7 CitationsSource
#2Mohammad JavanH-Index: 18
Last. Mahdi OroojiH-Index: 14
view all 5 authors...
Telemedicine refers to the use of information and communication technology to assist with medical information and services. In health care applications, high reliable communication links between the health care provider and the desired destination in the human body play a central role in designing end-to-end (E2E) telemedicine system. In the advanced health care applications, \text{e.g.}drug delivery, molecular communication becomes a major building block in bio-nano-medical applications. In ...