CRISPR/Cas9 Genome Editing for Tissue‐Specific In Vivo Targeting: Nanomaterials and Translational Perspective

Sahel, Deepak Kumar; Vora, Lalitkumar K.; Saraswat, Aishwarya; Sharma, Saurabh; Monpara, Jasmin; D’Souza, Anisha A.; Mishra, Deepakkumar; Tryphena, Kamatham Pushpa; Kawakita, Satoru; Khan, Shahid Ullah; Azhar, Mohd; Khatri, Dharmendra Kumar; Patel, Ketan; Thakur, Raghu

doi:10.1002/advs.202207512

Cited by 24 publications

(5 citation statements)

References 265 publications

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“…They discovered that RNPs exhibited an increased medicinal impact when delivered in EVs as opposed to RNPs alone and that the exosomes’ capacity to target liver cells improved tissue selectivity. This study demonstrated that exosomes from LX-2 cell lines might be used in liver tumor therapies ( Ding et al, 2021 ; Wan et al, 2022 ; Sahel et al, 2023 ).…”

Section: Need For Biomaterials: Advantages Over Other Delivery Methodsmentioning

confidence: 81%

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: 81%

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

Dubey,

Mostafavi

2023

Front. Chem.

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“…According to the above mentioned, CRISPR/Cas9 has brought great hope to overcome the dilemma of liver cancer treatment by directly editing the genes of immune checkpoints or indirectly affecting the expression of immune checkpoints by editing other genes. However, how to implement the strategy of accurately delivering CRISPR/Cas9 components in order to safely and efficiently take its advantages is an interesting but urgent problem to be solved [130,131]. Therefore, we summarize the method of delivering the system in these studies on the immune checkpoint of the system acting on liver cancer, so as to provide reference for future researchers to achieve accurate and safe delivery of the system for immunotherapy [120] with permission from BMJ Publishing Group Ltd. (B) In MAT2A-KO tumors, the portions of CD8+ T cells positive for immune checkpoint markers are significantly depleted.…”

Section: Delivery Strategies Of the Crispr/cas9 On Immune Checkpoints...mentioning

confidence: 99%

Current application and future perspective of CRISPR/cas9 gene editing system mediated immune checkpoint for liver cancer treatment

Tong,

Hu,

et al. 2024

Nanotechnology

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Liver cancer, which is well-known to us as one of human most prevalent malignancies across the globe, poses a significant risk to live condition and life safety of individuals in every region of the planet. It has been shown that immune checkpoint treatment may enhance survival benefits and make a significant contribution to patient prognosis, which makes it a promising and popular therapeutic option for treating liver cancer at the current time. However, there are only a very few numbers of patients who can benefit from the treatment and there also exist adverse events such as toxic effects and so on, which is still required further research and discussion. Fortunately, the clustered regularly interspaced short palindromic repeat/CRISPR-associated nuclease 9 (CRISPR/Cas9) provides a potential strategy for immunotherapy and immune checkpoint therapy of liver cancer. In this review, we focus on elucidating the fundamentals of the recently developed CRISPR/Cas9 technology as well as the present-day landscape of immune checkpoint treatment which pertains to liver cancer. What’s more, we aim to explore the molecular mechanism of immune checkpoint treatment in liver cancer based on CRISPR/Cas9 technology. At last, its encouraging and powerful potential in the future application of the clinic is discussed, along with the issues that already exist and the difficulties that must be overcome. To sum it all up, our ultimate goal is to create a fresh knowledge that we can utilize this new CRISPR/Cas9 technology for the current popular immune checkpoint therapy to overcome the treatment issues of liver cancer.

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“…This streamlines patient enrollment, study design, and personalized treatment [ 162 , 163 , 164 ]. AI has the potential to significantly enhance research, diagnostics, and therapeutics in the fields of exosomes, CAR T-cell therapy, and CRISPR/Cas9 [ 164 , 165 , 166 ].…”

Section: Ai For Drug Deliverymentioning

confidence: 99%

“…This streamlines patient enrollment, study design, and personalized treatment [162][163][164]. AI has the potential to significantly enhance research, diagnostics, and therapeutics in the fields of exosomes, CAR T-cell therapy, and CRISPR/Cas9 [164][165][166]. By utilizing AI's capabilities in data analysis, pattern recognition, and predictive modeling, the development of protein/peptide and gene therapy biologics can be accelerated, and the design and optimization of therapeutic molecules can be more efficient and targeted.…”

Section: Ai Tools For Biologics Product Developmentmentioning

confidence: 99%

Artificial Intelligence in Pharmaceutical Technology and Drug Delivery Design

Vora,

Gholap,

Jetha

et al. 2023

Pharmaceutics

Self Cite

242

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Artificial intelligence (AI) has emerged as a powerful tool that harnesses anthropomorphic knowledge and provides expedited solutions to complex challenges. Remarkable advancements in AI technology and machine learning present a transformative opportunity in the drug discovery, formulation, and testing of pharmaceutical dosage forms. By utilizing AI algorithms that analyze extensive biological data, including genomics and proteomics, researchers can identify disease-associated targets and predict their interactions with potential drug candidates. This enables a more efficient and targeted approach to drug discovery, thereby increasing the likelihood of successful drug approvals. Furthermore, AI can contribute to reducing development costs by optimizing research and development processes. Machine learning algorithms assist in experimental design and can predict the pharmacokinetics and toxicity of drug candidates. This capability enables the prioritization and optimization of lead compounds, reducing the need for extensive and costly animal testing. Personalized medicine approaches can be facilitated through AI algorithms that analyze real-world patient data, leading to more effective treatment outcomes and improved patient adherence. This comprehensive review explores the wide-ranging applications of AI in drug discovery, drug delivery dosage form designs, process optimization, testing, and pharmacokinetics/pharmacodynamics (PK/PD) studies. This review provides an overview of various AI-based approaches utilized in pharmaceutical technology, highlighting their benefits and drawbacks. Nevertheless, the continued investment in and exploration of AI in the pharmaceutical industry offer exciting prospects for enhancing drug development processes and patient care.

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CRISPR/Cas9 Genome Editing for Tissue‐Specific In Vivo Targeting: Nanomaterials and Translational Perspective

Cited by 24 publications

References 265 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

Current application and future perspective of CRISPR/cas9 gene editing system mediated immune checkpoint for liver cancer treatment

Artificial Intelligence in Pharmaceutical Technology and Drug Delivery Design

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