CRISPR/Cas9 systems: Delivery technologies and biomedical applications

Du, Yimin; Liu, Yanfei; Hu, Jiaxin; Peng, Xingxing; Liu, Zhenbao

doi:10.1016/j.ajps.2023.100854

Cited by 20 publications

(8 citation statements)

References 225 publications

(267 reference statements)

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“…Off-target effects are a limitation of CRISPR/Cas9 technology and are considered a significant risk in in vivo gene therapy ( Du et al, 2023 ). These effects occur when Cas9 proteins bind to PAM-like sequences or when guide RNAs (gRNAs) bind to unexpected nucleotide sequences at the target site.…”

Section: Limitations Of Crispr/cas9 In Disease Treatment and Applicationmentioning

confidence: 99%

“…The immune system’s reaction triggered by the Cas9 protein or vector in the host represents a significant challenge in the application of CRISPR/Cas9 technology ( Du et al, 2023 ). The Cas9 protein, originating from Streptococcus pyogenes, is considered an external antigen that can provoke an immune response upon introduction into the body ( Charlesworth et al, 2019 ).…”

Section: Limitations Of Crispr/cas9 In Disease Treatment and Applicationmentioning

confidence: 99%

“…While extreme immune responses to Cas9 are not extensively documented, the presence of Cas9 antibodies in humans indicates the potential exacerbation of hazards associated with CRISPR/Cas9 based gene therapy ( Li et al, 2021b ). Researchers are increasingly focusing on the immunogenicity caused by vectors, particularly viral vectors, as humans may have been previously exposed to them ( Du et al, 2023 ). Thus, the immune response generated by the CRISPR/Cas9 gene editing framework constitutes a primary detrimental factor in the context of in vivo gene therapy using CRISPR technology.…”

Section: Limitations Of Crispr/cas9 In Disease Treatment and Applicationmentioning

confidence: 99%

See 2 more Smart Citations

Application and perspective of CRISPR/Cas9 genome editing technology in human diseases modeling and gene therapy

Zhang,

Li,

Jin

2024

Front. Genet.

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The Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) mediated Cas9 nuclease system has been extensively used for genome editing and gene modification in eukaryotic cells. CRISPR/Cas9 technology holds great potential for various applications, including the correction of genetic defects or mutations within the human genome. The application of CRISPR/Cas9 genome editing system in human disease research is anticipated to solve a multitude of intricate molecular biology challenges encountered in life science research. Here, we review the fundamental principles underlying CRISPR/Cas9 technology and its recent application in neurodegenerative diseases, cardiovascular diseases, autoimmune related diseases, and cancer, focusing on the disease modeling and gene therapy potential of CRISPR/Cas9 in these diseases. Finally, we provide an overview of the limitations and future prospects associated with employing CRISPR/Cas9 technology for diseases study and treatment.

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Section: Limitations Of Crispr/cas9 In Disease Treatment and Applicationmentioning

confidence: 99%

Section: Limitations Of Crispr/cas9 In Disease Treatment and Applicationmentioning

confidence: 99%

Section: Limitations Of Crispr/cas9 In Disease Treatment and Applicationmentioning

confidence: 99%

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Application and perspective of CRISPR/Cas9 genome editing technology in human diseases modeling and gene therapy

Zhang,

Li,

Jin

2024

Front. Genet.

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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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“…To identify the precise genetic mutations causing cancer and understand their mechanisms, CRISPR/Cas9 gene editing for tumor cells and immune cells holds great potential in addition to traditional therapeutic approaches like surgery, radiotherapy, chemotherapy, and immunotherapy. Because of the genetic precision that CRISPR/Cas9 provides, treating cancers effectively may now be approached from a different perspective 20,21…”

Section: Applications In Biotechnology Medicine and Environmental Sci...mentioning

confidence: 99%

Advances in CRISPR-based technologies for genome editing in microorganisms

Chaudhari,

Ranjan

2024

IJMMTD

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CRISPR-based technologies have revolutionized the field of molecular biology by providing an unprecedented level of accuracy and efficiency in genome editing. CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a revolutionary gene-editing technology that allows precise modification of DNA in living organisms. It utilizes a guiding RNA molecule to target specific genes, enabling both gene knockout and insertion. CRISPR technology holds immense potential for applications in medicine, agriculture, and various scientific fields. This comprehensive review delves into the recent advancements made in CRISPR-based genome editing techniques, with a particular focus on their customized implementation for microorganisms. Starting with an examination of the history of CRISPR, the paper offers a detailed understanding of the major breakthroughs, complex challenges, and the wide range of potential applications associated with manipulating the genetic composition of bacteria, fungi, and other microbial entities.

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CRISPR/Cas9 systems: Delivery technologies and biomedical applications

Cited by 20 publications

References 225 publications

Application and perspective of CRISPR/Cas9 genome editing technology in human diseases modeling and gene therapy

Application and perspective of CRISPR/Cas9 genome editing technology in human diseases modeling and gene therapy

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

Advances in CRISPR-based technologies for genome editing in microorganisms

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