Genome editing of immune cells using CRISPR/Cas9

Kim, Segi; Hupperetz, Cedric; Lim, Seongjoon; Kim, Chan Hyuk

doi:10.5483/bmbrep.2021.54.1.245

Cited by 9 publications

(5 citation statements)

References 107 publications

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“…These observations suggest an Allele Sail can be a promising tool for population modification. We note that editors could also be used to alter the genomes of mitochondria and chloroplasts 58 , so as to tune interactions between components encoded by nuclear and organelle genomes that determine fitness 59 . Here we focused on nuclear genome editing.…”

Section: Discussionmentioning

confidence: 99%

Allele Sails: launching traits and fates into wild populations with DNA sequence modifiers

Johnson,

Hay,

Maselko

2024

Preprint

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Population-scale genome editing can be used to alter the composition or fate of wild populations. One approach to achieving these aims utilizes a synthetic gene drive element a multi-gene cassette-to bring about an increase in the frequency of an existing allele. However, the use of gene drives is complicated by the multiple scientific, regulatory, and social issues associated with transgene persistence and gene flow. Alternatives in which transgenes are not driven could potentially avoid some of these issues. Here we propose an approach to population scale gene editing using a system we refer to as an Allele Sail. An Allele Sail consists of a genome editor (the Wind) that introduces DNA sequence edits (the Sail) at one or more sites, resulting in progeny that are viable and fertile. The editor, such as a sequence-specific nuclease, or a prime- or base-editor, is inherited in a Mendelian fashion. Meanwhile, the edits it creates experience a Super-Mendelian increase in frequency. We explore this system using agent-based modeling, and identify contexts in which a single, low frequency release of an editor brings edits to a very high frequency. We also identify conditions in which manipulation of sex determination can be used to bring about population suppression. Current regulatory frameworks often distinguish between transgenics as genetically modified organisms (GMOs), and their edited non-transgenic progeny as non-GMO. In this context an Allele Sail provides a path to alter traits and fates of wild populations in ways that may be considered more acceptable.

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Section: Discussionmentioning

confidence: 99%

Allele Sails: launching traits and fates into wild populations with DNA sequence modifiers

Johnson,

Hay,

Maselko

2024

Preprint

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“…6 ). In addition, when Cas9-induced DSBs form, HDR can occur as an alternative pathway to NHEJ in the presence of sister chromatids ( 30 , 31 ). RAD51 coils around the 3’ resected ssDNA of the DSB to create nucleoprotein filaments.…”

Section: Discussionmentioning

confidence: 99%

Elevated expression of exogenous RAD51 enhances the CRISPR/Cas9-mediated genome editing efficiency

Park

Yoon

Choi

et al. 2023

BMB Rep

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Genome editing using CRISPR-associated technology is widely used to modify the genomes rapidly and efficiently on specific DNA double-strand breaks (DSBs) induced by Cas9 endonuclease. However, despite swift advance in Cas9 engineering, structural basis of Cas9-recognition and cleavage complex remains unclear. Proper assembly of this complex correlates to effective Cas9 activity, leading to high efficacy of genome editing events. Here, we develop a CRISPR/Cas9-RAD51 plasmid constitutively expressing RAD51, which can bind to singlestranded DNA for DSB repair. We show that the efficiency of CRISPR-mediated genome editing can be significantly improved by expressing RAD51, responsible for DSB repair via homologous recombination (HR), in both gene knock-out and knock-in processes. In cells with CRISPR/Cas9-RAD51 plasmid, expression of the target genes (cohesin SMC3 and GAPDH) was reduced by more than 1.9-fold compared to the CRISPR/Cas9 plasmid for knock-out of genes. Furthermore, CRISPR/Cas9-RAD51 enhanced the knock-in efficiency of DsRed donor DNA. Thus, the CRISPR/Cas9-RAD51 system is useful for applications requiring precise and efficient genome edits not accessible to HR-deficient cell genome editing and for developing CRISPR/Cas9-mediated knockout technology.

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“…CRISPR/Cas9 technology can be used to change the genomes of immune and stem cells used in tissue engineering to improve their therapeutic potential. For instance, T cells may be modified using CRISPR/Cas9 to form chimeric antigen receptors (CARs), which can enhance their ability to detect and destroy cancer cells ( Gao et al, 2019 ; Kim et al, 2021 ). Overall, the potential of CRISPR/Cas9 in tissue engineering is vast, and ongoing research is likely to uncover new ways to harness this powerful technology to create functional tissues and organs that can be used to treat a wide range of diseases and injuries.…”

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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Genome editing of immune cells using CRISPR/Cas9

Cited by 9 publications

References 107 publications

Allele Sails: launching traits and fates into wild populations with DNA sequence modifiers

Allele Sails: launching traits and fates into wild populations with DNA sequence modifiers

Elevated expression of exogenous RAD51 enhances the CRISPR/Cas9-mediated genome editing efficiency

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

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