CRISPR/Cas9 Plasmid Delivery Through the CPP: PepFect14

Falato, Luca; Vunk, Birgit; Langel, Ülo

doi:10.1007/978-1-0716-1752-6_38

Cited by 5 publications

(3 citation statements)

References 14 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Another delivery approach is by using Cas9 protein fused with sgRNA (together, the Cas9-RNP) using synthetic delivery vehicles, transient, direct pathway for introduction of the [ 138 ]. Furthermore, a group developed a plasmid delivery approach for CRISPR-Cas9 group by the characteristics of the Cell-Penetrating Peptide (PepFect14) in a Bomirsky Hamster Melanoma cell line aiming to deactivate the luciferase gene and to express Green Fluorescent Protein, and by labeling the CRISPR plasmid with Cy5-ensured screening of the cellular entrance through fluorescent microscopy [ 139 ]. Moreover, NSC and MSC-based deliveries are mentioned earlier in this review.…”

Section: Crispr-cas Deliverymentioning

confidence: 99%

Systems Medicine for Precise Targeting of Glioblastoma

Zeng¹,

Zeng

2023

Mol Biotechnol

View full text Add to dashboard Cite

Glioblastoma (GBM) is a malignant cancer that is fatal even after standard therapy and the effects of current available therapeutics are not promising due its complex and evolving epigenetic and genetic profile. The mysteries that lead to GBM intratumoral heterogeneity and subtype transitions are not entirely clear. Systems medicine is an approach to view the patient in a whole picture integrating systems biology and synthetic biology along with computational techniques. Since the GBM oncogenesis involves genetic mutations, various therapies including gene therapeutics based on CRISPR-Cas technique, MicroRNAs, and implanted synthetic cells endowed with synthetic circuits against GBM with neural stem cells and mesenchymal stem cells acting as potential vehicles carrying therapeutics via the intranasal route, avoiding the risks of invasive methods in order to reach the GBM cells in the brain are discussed and proposed in this review. Systems medicine approach is a rather novel strategy, and since the GBM of a patient is complex and unique, thus to devise an individualized treatment strategy to tailor personalized multimodal treatments for the individual patient taking into account the phenotype of the GBM, the unique body health profile of the patient and individual responses according to the systems medicine concept might show potential to achieve optimum effects.

show abstract

Section: Crispr-cas Deliverymentioning

confidence: 99%

Systems Medicine for Precise Targeting of Glioblastoma

Zeng¹,

Zeng

2023

Mol Biotechnol

View full text Add to dashboard Cite

show abstract

“…Furthermore, inorganic nanovehicles aid precise targeted delivery of CRISPR/Cas cargo for in vivo therapy. The next-generation single-chain antibodies [ 23 , 233 ], antigen-binding fragments [ 23 ], aptamers (single-stranded oligonucleotides) [ 235 ], AS1411 (G rich DNA oligonucleotide) [ 324 ], and peptides [ 55 ] can be easefully conjugated on the polymer-coated surface of nanovehicles, which will promote targeting in TNBC. After successful internalization of CRISPR-nano complex via endocytosis, the complex face acidic environment and multiple catalytic enzymes of endosome that can inactive CRISPR/Cas components.…”

Section: Design Strategies For Effective Crispr-nano Engineering In Tnbcmentioning

confidence: 99%

“…The encapsulation via nano-based cargo precludes the susceptible CRISPR complex from deterioration intervened by proteases and nucleases in biological fluids matrix [ 51 ]. Since nucleic acid has a negative charge, besides the Cas9 nuclease's immense size (≈160 kDa), due to this nanocarriers are designed with positive charges to enhance the packaging capacity and effective communication with cells [ [52] , [53] , [54] ], or festooned with cell-penetrating peptides (CPPs) [ 30 , 55 ] to ease the plasma membrane penetration. However, its successful clinical application is a tremendous task due to low in viv o efficacy of nanocarriers [ 41 ].…”

Section: Introductionmentioning

confidence: 99%

Nanomaterial-assisted CRISPR gene-engineering – A hallmark for triple-negative breast cancer therapeutics advancement

Farheen¹,

Hosmane²,

Zhao³

et al. 2022

Materials Today Bio

View full text Add to dashboard Cite

The menace of severe adverse events and deaths associated with viral gene therapy and its potential solution

Kachanov,

Kostyusheva,

Brezgin

et al. 2024

Medicinal Research Reviews

View full text Add to dashboard Cite

Over the past decade, in vivo gene replacement therapy has significantly advanced, resulting in market approval of numerous therapeutics predominantly relying on adeno‐associated viral vectors (AAV). While viral vectors have undeniably addressed several critical healthcare challenges, their clinical application has unveiled a range of limitations and safety concerns. This review highlights the emerging challenges in the field of gene therapy. At first, we discuss both the role of biological barriers in viral gene therapy with a focus on AAVs, and review current landscape of in vivo human gene therapy. We delineate advantages and disadvantages of AAVs as gene delivery vehicles, mostly from the safety perspective (hepatotoxicity, cardiotoxicity, neurotoxicity, inflammatory responses etc.), and outline the mechanisms of adverse events in response to AAV. Contribution of every aspect of AAV vectors (genomic structure, capsid proteins) and host responses to injected AAV is considered and substantiated by basic, translational and clinical studies. The updated evaluation of recent AAV clinical trials and current medical experience clearly shows the risks of AAVs that sometimes overshadow the hopes for curing a hereditary disease. At last, a set of established and new molecular and nanotechnology tools and approaches are provided as potential solutions for mitigating or eliminating side effects. The increasing number of severe adverse reactions and, sadly deaths, demands decisive actions to resolve the issue of immune responses and extremely high doses of viral vectors used for gene therapy. In response to these challenges, various strategies are under development, including approaches aimed at augmenting characteristics of viral vectors and others focused on creating secure and efficacious non‐viral vectors. This comprehensive review offers an overarching perspective on the present state of gene therapy utilizing both viral and non‐viral vectors.

show abstract

CRISPR/Cas9 Plasmid Delivery Through the CPP: PepFect14

Cited by 5 publications

References 14 publications

Systems Medicine for Precise Targeting of Glioblastoma

Systems Medicine for Precise Targeting of Glioblastoma

Nanomaterial-assisted CRISPR gene-engineering – A hallmark for triple-negative breast cancer therapeutics advancement

The menace of severe adverse events and deaths associated with viral gene therapy and its potential solution

Contact Info

Product

Resources

About