Poly-sgRNA/siRNA ribonucleoprotein nanoparticles for targeted gene disruption

Ha, Jong Seong; Lee, Jae Sung; Jeong, Jaepil; Kim, Hejin; Byun, Ji Young; Kim, Sang Ah; Lee, Hee Jae; Chung, Hak Suk; Lee, Jong Bum; Ahn, Dae‐Ro

doi:10.1016/j.jconrel.2017.02.007

Cited by 39 publications

(30 citation statements)

References 29 publications

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“…In order to address these challenges, transient delivery of sgRNA along with the Cas9 mRNA or Cas9 protein has been achieved using non-viral methods such as electroporation, 15,16 hydrodynamic injection, 17 microinjection, 18 lipids, [19][20][21][22] peptides, [23][24][25][26][27][28] polyethylenimine in combination with other agents such as DNA nanoclew, graphene oxide, cholesterol, [29][30][31] gold nanoparticles and other nanostructures, [32][33][34][35][36][37][38] extracellular vesicles, [39][40][41][42] virus-like particles, 43 and biolistic delivery in plants. 44,45 Apart from these methods, many hybrid frameworks have also been used to deliver CRISPR/Cas9.…”

Section: Introductionmentioning

confidence: 99%

Non-viral delivery of CRISPR/Cas9 complex using CRISPR-GPS nanocomplexes

Jain

Rananaware

et al. 2019

Nanoscale

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Section: Introductionmentioning

confidence: 99%

Non-viral delivery of CRISPR/Cas9 complex using CRISPR-GPS nanocomplexes

Jain

Rananaware

et al. 2019

Nanoscale

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“…In particular, when shaped into nanoparticles, RCA products would have great potential to be utilized in biomedical applications, such as carriers for drug delivery, as DNA and RNA are highly biocompatible materials. Cationic lipids and polymeric amines have been widely used for nano-formulation of RCA products and have allowed for the improvement of some critical properties, such as a high cellular uptake efficiency and the nuclease resistance required for successful applications in vitro and in vivo [ 5 , 7 , 8 , 9 , 10 ]. However, as cationic lipids and polymeric amines may compromise the biocompatibility of DNA and RNA materials due to their potential cytotoxicity [ 11 , 12 ], the nanoparticle formation in a self-assembled manner without external additives is a more valid approach, especially when cellular and in vivo applications are considered.…”

Section: Introductionmentioning

confidence: 99%

Shaping Rolling Circle Amplification Products into DNA Nanoparticles by Incorporation of Modified Nucleotides and Their Application to In Vitro and In Vivo Delivery of a Photosensitizer

et al. 2018

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Rolling circle amplification (RCA) is a robust way to generate DNA constructs, which are promising materials for biomedical applications including drug delivery because of their high biocompatibility. To be employed as a drug delivery platform, however, the DNA materials produced by RCA need to be shaped into nanoparticles that display both high cellular uptake efficiency and nuclease resistance. Here, we showed that the DNA nanoparticles (DNPs) can be prepared with RCA and modified nucleotides that have side-chains appended on the nucleobase are capable of interacting with the DNA strands of the resulting RCA products. The incorporation of the modified nucleotides improved cellular uptake efficiency and nuclease resistance of the DNPs. We also demonstrated that these DNPs could be employed as carriers for the delivery of a photosensitizer into cancer cells to achieve photodynamic therapy upon irradiation at both the in vitro and in vivo levels.

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“…In the iTOP method, macromolecules are delivered into cells via micropinocytosis and using propanebetaine with minimal toxicity ( 198 ). In addition, Ha et al have presented an innovative way of delivery through Poly-sgRNA/siRNA nanoparticles, in order to disrupt gene expression ( 199 ). The principle in using these nanoparticles is based on the assembly of siRNA (substrate of Dicer) and sgRNAs, which are cleaved through the action of Dicer following rolling circle transcription (RCT).…”

Section: Challenges Associated With the Crispr-based Gene Methods mentioning

confidence: 99%

“…The principle in using these nanoparticles is based on the assembly of siRNA (substrate of Dicer) and sgRNAs, which are cleaved through the action of Dicer following rolling circle transcription (RCT). The advantages are as follows: i) Minimal toxicity; ii) nanoparticle strength and stability; and iii) single administration of components to ensure the permanent disarrangement of target gene expression ( 199 ). Other delivery methods have been reported to be involved in the ' in vivo ' transfer of constituents of the CRISPR system.…”

Section: Challenges Associated With the Crispr-based Gene Methods mentioning

confidence: 99%

CRISPR therapeutic tools for complex genetic disorders and cancer (Review)

et al. 2018

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One of the fundamental discoveries in the field of biology is the ability to modulate the genome and to monitor the functional outputs derived from genomic alterations. In order to unravel new therapeutic options, scientists had initially focused on inducing genetic alterations in primary cells, in established cancer cell lines and mouse models using either RNA interference or cDNA overexpression or various programmable nucleases [zinc finger nucleases (ZNF), transcription activator-like effector nucleases (TALEN)]. Even though a huge volume of data was produced, its use was neither cheap nor accurate. Therefore, the clustered regularly interspaced short palindromic repeats (CRISPR) system was evidenced to be the next step in genome engineering tools. CRISPR-associated protein 9 (Cas9)-mediated genetic perturbation is simple, precise and highly efficient, empowering researchers to apply this method to immortalized cancerous cell lines, primary cells derived from mouse and human origins, xenografts, induced pluripotent stem cells, organoid cultures, as well as the generation of genetically engineered animal models. In this review, we assess the development of the CRISPR system and its therapeutic applications to a wide range of complex diseases (particularly distinct tumors), aiming at personalized therapy. Special emphasis is given to organoids and CRISPR screens in the design of innovative therapeutic approaches. Overall, the CRISPR system is regarded as an eminent genome engineering tool in therapeutics. We envision a new era in cancer biology during which the CRISPR-based genome engineering toolbox will serve as the fundamental conduit between the bench and the bedside; nonetheless, certain obstacles need to be addressed, such as the eradication of side-effects, maximization of efficiency, the assurance of delivery and the elimination of immunogenicity.

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Poly-sgRNA/siRNA ribonucleoprotein nanoparticles for targeted gene disruption

Cited by 39 publications

References 29 publications

Non-viral delivery of CRISPR/Cas9 complex using CRISPR-GPS nanocomplexes

Non-viral delivery of CRISPR/Cas9 complex using CRISPR-GPS nanocomplexes

Shaping Rolling Circle Amplification Products into DNA Nanoparticles by Incorporation of Modified Nucleotides and Their Application to In Vitro and In Vivo Delivery of a Photosensitizer

CRISPR therapeutic tools for complex genetic disorders and cancer (Review)

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