Recent advances in the delivery and applications of nonviral CRISPR/Cas9 gene editing

Sinclair, Frazer; Begum, Anjuman Ara; Dai, Charles C; Tóth, István; Moyle, Peter M.

doi:10.1007/s13346-023-01320-z

Cited by 20 publications

(6 citation statements)

References 144 publications

(141 reference statements)

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“… iv. Electroporation: To allow CRISPR/Cas9 components to enter cells, electroporation entails delivering electrical pulses to temporarily break apart the cell membrane ( Sinclair et al, 2023 ). Nucleic acids may be introduced into a variety of cells, including those that are challenging to transfect, using this standard technique.…”

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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“…Current nonviral delivery systems are limited by their loading efficiency, non-disease targeting, and problematic clearance out of the brain [ 973 ]. These problems are being solved one by one [ 960 , 962 , 974 ] and, although most of the aforementioned studies are in preclinical phases, these novel CRISPR-Cas9–brain delivery systems seem a versatile and potent platform for treating GB and other brain diseases.…”

Section: Nanotherapiesmentioning

confidence: 99%

Glioblastoma Therapy: Past, Present and Future

Obrador,

Moreno-Murciano,

Oriol-Caballo

et al. 2024

IJMS

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Glioblastoma (GB) stands out as the most prevalent and lethal form of brain cancer. Although great efforts have been made by clinicians and researchers, no significant improvement in survival has been achieved since the Stupp protocol became the standard of care (SOC) in 2005. Despite multimodality treatments, recurrence is almost universal with survival rates under 2 years after diagnosis. Here, we discuss the recent progress in our understanding of GB pathophysiology, in particular, the importance of glioma stem cells (GSCs), the tumor microenvironment conditions, and epigenetic mechanisms involved in GB growth, aggressiveness and recurrence. The discussion on therapeutic strategies first covers the SOC treatment and targeted therapies that have been shown to interfere with different signaling pathways (pRB/CDK4/RB1/P16ink4, TP53/MDM2/P14arf, PI3k/Akt-PTEN, RAS/RAF/MEK, PARP) involved in GB tumorigenesis, pathophysiology, and treatment resistance acquisition. Below, we analyze several immunotherapeutic approaches (i.e., checkpoint inhibitors, vaccines, CAR-modified NK or T cells, oncolytic virotherapy) that have been used in an attempt to enhance the immune response against GB, and thereby avoid recidivism or increase survival of GB patients. Finally, we present treatment attempts made using nanotherapies (nanometric structures having active anti-GB agents such as antibodies, chemotherapeutic/anti-angiogenic drugs or sensitizers, radionuclides, and molecules that target GB cellular receptors or open the blood–brain barrier) and non-ionizing energies (laser interstitial thermal therapy, high/low intensity focused ultrasounds, photodynamic/sonodynamic therapies and electroporation). The aim of this review is to discuss the advances and limitations of the current therapies and to present novel approaches that are under development or following clinical trials.

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“…CRISPR can be used to engineer immune cells to express chimeric antigen receptors (CARs) or enhance antipathogen effector functions, thereby increasing the efficacy of immunotherapeutic approaches against zoonotic diseases [108][109][110]. CRISPRmediated genome editing in animal models can facilitate the preclinical evaluation of immunotherapies, accelerate their application in clinical practice, and provide reliable efficacy information.…”

Section: The Use Of Crispr-cas9 In the Development Of Vaccines Tradit...mentioning

confidence: 99%

CRISPR-Cas9 Genome Editing Technology for Zoonotic Disease Control in Indonesia: A Comprehensive Review

Adnyana,

Eljatin,

Sudaryati

et al. 2024

JMHT

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The increasing emergence of zoonotic diseases originating from vectors, rodents, mammals, and others has increased the potential for outbreaks of pandemics in the Indonesian territory. Although control and prevention have been implemented, these efforts have not yet revealed a bright spot; therefore, elaboration with advanced CRISPR-Cas9 technology is a way to accelerate efforts to control zoonotic diseases in Indonesia. However, there is limited literature on this topic. This review aims to comprehensively describe, identify, and summarize the application of CRISPR-Cas9 genome-editing technology in zoonotic disease control in Indonesia. Our findings show that CRISPR-Cas9 genome editing technology offers an innovative approach for zoonotic disease control by targeting disease vectors, modifying animal reservoirs, improving disease surveillance, enhancing vaccine development, and exploring traditional medicine candidates and immunotherapy. The high level of precision, efficiency, and versatility in targeting genomes capable of disrupting, damaging, and disrupting the disease transmission cycle in pathogens makes CRISPR-Cas9 highly effective. However, challenges such as off-target impacts, regulatory complexity, and ethical considerations must be overcome with inter- and multidisciplinary collaboration to promote transparency, equity, and public engagement throughout the process of implementing this technology in the field, especially in Indonesia.

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Recent advances in the delivery and applications of nonviral CRISPR/Cas9 gene editing

Cited by 20 publications

References 144 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

Glioblastoma Therapy: Past, Present and Future

CRISPR-Cas9 Genome Editing Technology for Zoonotic Disease Control in Indonesia: A Comprehensive Review

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