Pre-clinical non-viral vectors exploited for<i>in vivo</i>CRISPR/Cas9 gene editing: an overview

Rouatbi, Nadia; McGlynn, Tasneem; Al‐Jamal, Khuloud T.

doi:10.1039/d1bm01452h

Cited by 14 publications

(9 citation statements)

References 154 publications

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“…Chitosan is often utilized as a cationic polymer to bind with the negatively charged CRISPR/Cas9 components in chitosan-based CRISPR/Cas9 delivery systems, which mainly include the creation of nanoparticles. Cells may quickly absorb the resultant nanoparticles and transfer them to the nucleus, where the CRISPR/Cas9 components can carry out their gene-editing task ( Givens et al, 2018 ; Zhang et al, 2018 ; Rouatbi et al, 2022 ). The CRISPR/Cas9 combination was loaded with CsNPs by researchers to treat pulmonary arterial hypertension (PAH).…”

Section: Need For Biomaterials: Advantages Over Other Delivery Methodsmentioning

confidence: 99%

Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy

Dubey,

Mostafavi

2023

Front. Chem.

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The use of biomaterials in delivering CRISPR/Cas9 for gene therapy in infectious diseases holds tremendous potential. This innovative approach combines the advantages of CRISPR/Cas9 with the protective properties of biomaterials, enabling accurate and efficient gene editing while enhancing safety. Biomaterials play a vital role in shielding CRISPR/Cas9 components, such as lipid nanoparticles or viral vectors, from immunological processes and degradation, extending their effectiveness. By utilizing the flexibility of biomaterials, tailored systems can be designed to address specific genetic diseases, paving the way for personalized therapeutics. Furthermore, this delivery method offers promising avenues in combating viral illnesses by precisely modifying pathogen genomes, and reducing their pathogenicity. Biomaterials facilitate site-specific gene modifications, ensuring effective delivery to infected cells while minimizing off-target effects. However, challenges remain, including optimizing delivery efficiency, reducing off-target effects, ensuring long-term safety, and establishing scalable production techniques. Thorough research, pre-clinical investigations, and rigorous safety evaluations are imperative for successful translation from the laboratory to clinical applications. In this review, we discussed how CRISPR/Cas9 delivery using biomaterials revolutionizes gene therapy and infectious disease treatment, offering precise and safe editing capabilities with the potential to significantly improve human health and quality of life.

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Section: Need For Biomaterials: Advantages Over Other Delivery Methodsmentioning

confidence: 99%

Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy

Dubey,

Mostafavi

2023

Front. Chem.

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“…To date, several types of nano-vectors, including lipids (such as 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine [DOPE] and cholesterol), polymers (such as polyethyleneimine, poly-L-lysine, and chitosan), inorganic chemicals (such as cationic arginine gold nanoparticles and CRISPR-Gold), and exosomes (such as exosome-liposome hybrid and engineered exosomes: CD9-HuR exosomes and NanoMEDIC) have been developed [ 70 , 71 , 72 ]. Since these nano-vectors can prevent the degradation of the CRISPR/Cas9 genome-edition system and directly enter into the nucleus to perform genome editing in vivo, they can be ideal delivery platforms for the CRISPR/Cas9 system, enabling effective ex vivo or in vivo transfection of CRISPR reagents encoding DNA, RNA, or RNP in a highly efficient and safe way [ 73 , 74 , 75 ]. For example, Wei et al [ 76 ] encapsulated RNPs with cationic lipids composed of 1,2-dioleoyl-3-(trimethylammonium) propane as permanent cationic supplements, DOPE as a helper lipid, and cholesterol as a sterol, postmodified with polyethylene glycol phospholipid as PEGylated lipids to yield nanoparticles with retained activity and redirect DNA editing to the target tissues with decreased clearance and immunogenicity.…”

Section: Limitations Of and Possibilities For In Utero Genome Editing...mentioning

confidence: 99%

Recent Genome-Editing Approaches toward Post-Implanted Fetuses in Mice

Nakamura

Inada

Saitoh

2023

BioTech

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Genome editing, as exemplified by the CRISPR/Cas9 system, has recently been employed to effectively generate genetically modified animals and cells for the purpose of gene function analysis and disease model creation. There are at least four ways to induce genome editing in individuals: the first is to perform genome editing at the early preimplantation stage, such as fertilized eggs (zygotes), for the creation of whole genetically modified animals; the second is at post-implanted stages, as exemplified by the mid-gestational stages (E9 to E15), for targeting specific cell populations through in utero injection of viral vectors carrying genome-editing components or that of nonviral vectors carrying genome-editing components and subsequent in utero electroporation; the third is at the mid-gestational stages, as exemplified by tail-vein injection of genome-editing components into the pregnant females through which the genome-editing components can be transmitted to fetal cells via a placenta-blood barrier; and the last is at the newborn or adult stage, as exemplified by facial or tail-vein injection of genome-editing components. Here, we focus on the second and third approaches and will review the latest techniques for various methods concerning gene editing in developing fetuses.

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“…For example, CRISPR/Cas9 gene knockout vectors are usually constructed and transferred into plants. After screening for one or two generations, target-gene knockout mutants may be obtained [ 24 ]. One target gene can be knocked out or multiple target genes can be knocked out simultaneously.…”

Section: Crispr/cas9-based Gene-editing Toolsmentioning

confidence: 99%

CRISPR/Cas9 Technology and Its Utility for Crop Improvement

Liu

Chen

et al. 2022

IJMS

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The rapid growth of the global population has resulted in a considerable increase in the demand for food crops. However, traditional crop breeding methods will not be able to satisfy the worldwide demand for food in the future. New gene-editing technologies, the most widely used of which is CRISPR/Cas9, may enable the rapid improvement of crop traits. Specifically, CRISPR/Cas9 genome-editing technology involves the use of a guide RNA and a Cas9 protein that can cleave the genome at specific loci. Due to its simplicity and efficiency, the CRISPR/Cas9 system has rapidly become the most widely used tool for editing animal and plant genomes. It is ideal for modifying the traits of many plants, including food crops, and for creating new germplasm materials. In this review, the development of the CRISPR/Cas9 system, the underlying mechanism, and examples of its use for editing genes in important crops are discussed. Furthermore, certain limitations of the CRISPR/Cas9 system and potential solutions are described. This article will provide researchers with important information regarding the use of CRISPR/Cas9 gene-editing technology for crop improvement, plant breeding, and gene functional analyses.

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Pre-clinical non-viral vectors exploited forin vivoCRISPR/Cas9 gene editing: an overview

Abstract: Non-viral delivery technologies for efficient in vivo Clustered Regulatory Interspaced Short Palindromic Repeats (CRISPR/Cas9) gene editing.

Cited by 14 publications

References 154 publications

Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy

Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy

Recent Genome-Editing Approaches toward Post-Implanted Fetuses in Mice

CRISPR/Cas9 Technology and Its Utility for Crop Improvement

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