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

Jain, Piyush; Lo, Justin H.; Rananaware, Santosh R.; Downing, Marco A.; Panda, Apekshya; Tai, Michelle; Raghavan, Srivatsan; Fleming, Heather E.; Bhatia, Sangeeta N.

doi:10.1039/c9nr01786k

Cited by 38 publications

(42 citation statements)

References 61 publications

(71 reference statements)

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“…The iRGD-containing tandem lipopeptide, palmitoyl-TP-iRGD, was co-assembled with Cas9 RNP for targeted RNP delivery (Figure 16 A). The targeted lipopeptide exhibited higher efficiencies than Lipofectamine RNAi MAX in various cell lines 191 . In a separate study, nanoparticles consisting of PEI-CD and PEI-AD were used for RNP delivery and the particles were further coated with DOTAP lipids bearing two peptides 279 .…”

Section: Targeted Delivery Systems For Rnp Deliverymentioning

confidence: 99%

“…The caprylic acid-modified peptide C8dNP2 exhibited the highest ability to form homologous nanosomes, and efficiently delivered HypaCas9 RNP into HEK and GBM cells with efficiencies higher than Lipofectamine 2000 and CRISPR MAX 189 . In another study, modification of lipopeptides with targeting peptides enabled cell-selective gene editing 191 .…”

Section: Materials For Rnp Deliverymentioning

confidence: 99%

See 1 more Smart Citation

Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing

Song¹,

Shen²,

Li³

et al. 2021

Theranostics

253

184

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CRISPR/Cas9 genome editing has gained rapidly increasing attentions in recent years, however, the translation of this biotechnology into therapy has been hindered by efficient delivery of CRISPR/Cas9 materials into target cells. Direct delivery of CRISPR/Cas9 system as a ribonucleoprotein (RNP) complex consisting of Cas9 protein and single guide RNA (sgRNA) has emerged as a powerful and widespread method for genome editing due to its advantages of transient genome editing and reduced off-target effects. In this review, we summarized the current Cas9 RNP delivery systems including physical approaches and synthetic carriers. The mechanisms and beneficial roles of these strategies in intracellular Cas9 RNP delivery were reviewed. Examples in the development of stimuli-responsive and targeted carriers for RNP delivery are highlighted. Finally, the challenges of current Cas9 RNP delivery systems and perspectives in rational design of next generation materials for this promising field will be discussed.

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Section: Targeted Delivery Systems For Rnp Deliverymentioning

confidence: 99%

Section: Materials For Rnp Deliverymentioning

confidence: 99%

Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing

Song¹,

Shen²,

Li³

et al. 2021

Theranostics

253

184

View full text Add to dashboard Cite

show abstract

“…These siRNA loaded TPNs entered in the cytosol via a receptor-specific fashion and could be used to target ovarian cancer. Furthermore, the slightly modified TPNs, by changing the Lyp-1 targeting sequence with iRGD, were demonstrated to deliver siRNA to pancreatic cancer [119], as well as single chimeric guide (sgRNA)/Cas9 protein complex inside cells [120].…”

Section: Cell/organ Targetingmentioning

confidence: 99%

Peptide-Based Nanoparticles for Therapeutic Nucleic Acid Delivery

et al. 2021

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Gene therapy offers the possibility to skip, repair, or silence faulty genes or to stimulate the immune system to fight against disease by delivering therapeutic nucleic acids (NAs) to a patient. Compared to other drugs or protein treatments, NA-based therapies have the advantage of being a more universal approach to designing therapies because of the versatility of NA design. NAs (siRNA, pDNA, or mRNA) have great potential for therapeutic applications for an immense number of indications. However, the delivery of these exogenous NAs is still challenging and requires a specific delivery system. In this context, beside other non-viral vectors, cell-penetrating peptides (CPPs) gain more and more interest as delivery systems by forming a variety of nanocomplexes depending on the formulation conditions and the properties of the used CPPs/NAs. In this review, we attempt to cover the most important biophysical and biological aspects of non-viral peptide-based nanoparticles (PBNs) for therapeutic nucleic acid formulations as a delivery system. The most relevant peptides or peptide families forming PBNs in the presence of NAs described since 2015 will be presented. All these PBNs able to deliver NAs in vitro and in vivo have common features, which are characterized by defined formulation conditions in order to obtain PBNs from 60 nm to 150 nm with a homogeneous dispersity (PdI lower than 0.3) and a positive charge between +10 mV and +40 mV.

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“…Several examples of myristoyl–transportan transfection are available: NPs incorporating myristoyl–transportan and tumor-homing peptides carrying siRNA, a CpG DNA ligand of TLR9 suppressed tumor growth in several animal models of various cancers after systemic intravenous (i.v.) administration [ 98 ]; myristoyl–transportan conjugated to a transferrin receptor-targeting peptide (myr-TP-Tf) encapsulating siRNA targeted it to the brain with a functional gene silencing effect in a human glioma [ 99 ]; and a nanocomplex based on a tandem peptide of myristoyl–transportan and Lyp-1 showed the internalization of sgRNA/Cas9 ribonucleoprotein complexes and genome editing in cell lines [ 100 ].…”

Section: Transportanmentioning

confidence: 99%

Cell-Penetrating Peptides and Transportan

Langel

2021

Pharmaceutics

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In the most recent 25–30 years, multiple novel mechanisms and applications of cell-penetrating peptides (CPP) have been demonstrated, leading to novel drug delivery systems. In this review, I present a brief introduction to the CPP area with selected recent achievements. This is followed by a nostalgic journey into the research in my own laboratories, which lead to multiple CPPs, starting from transportan and paving a way to CPP-based therapeutic developments in the delivery of bio-functional materials, such as peptides, proteins, vaccines, oligonucleotides and small molecules, etc.

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

Abstract: A tandem peptide-lipid based nanocomplexation strategy for targeted delivery of CRISPR/Cas9 in cells for efficient gene editing.

Cited by 38 publications

References 61 publications

Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing

Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing

Peptide-Based Nanoparticles for Therapeutic Nucleic Acid Delivery

Cell-Penetrating Peptides and Transportan

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