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

Abstract

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.

The key role of miRNA in syndromic and sporadic forms of ascending aortic aneurysms as biomarkers and targets of novel therapeutic strategies

“…Based on the evidence reported above, we propose that a strong genetic editing technique might be a solution for reversing the onset and progression of familial forms of ATAA, as well as the integration of gene-editing techniques with nanotechnology ( Dong et al, 2022 ; Testa and Musunuru, 2023 ). Therefore, the CRISPR/Cas9 system and techniques involved in the chemical modification of RNAs are emerging, and are also being incorporated into nanoparticles (NPs) for studying diseases ( Dubey and Mostafavi, 2023 ), such as ATAA, and/or investigating potential disease corrections through restoration ( Testa and Musunuru, 2023 ). The CRISPR/Cas9 system is substantially applied for both editing the genome of zygotes, thereby generating genetically modified animal species, and treating human diseases such as aneurysms ( Dubey and Mostafavi, 2023 ).…”

“…Therefore, the CRISPR/Cas9 system and techniques involved in the chemical modification of RNAs are emerging, and are also being incorporated into nanoparticles (NPs) for studying diseases ( Dubey and Mostafavi, 2023 ), such as ATAA, and/or investigating potential disease corrections through restoration ( Testa and Musunuru, 2023 ). The CRISPR/Cas9 system is substantially applied for both editing the genome of zygotes, thereby generating genetically modified animal species, and treating human diseases such as aneurysms ( Dubey and Mostafavi, 2023 ). Furthermore, the development of cautious and efficient treatments is developing thanks to releasing of the CRISPR/Cas9 system into body tissues and targeting specific cells, including ECs or VSMCS ( Dubey and Mostafavi, 2023 ).…”

“…The CRISPR/Cas9 system is substantially applied for both editing the genome of zygotes, thereby generating genetically modified animal species, and treating human diseases such as aneurysms ( Dubey and Mostafavi, 2023 ). Furthermore, the development of cautious and efficient treatments is developing thanks to releasing of the CRISPR/Cas9 system into body tissues and targeting specific cells, including ECs or VSMCS ( Dubey and Mostafavi, 2023 ).…”

“…Data in the literature suggest several types of delivery systems that enable genome editing. A delivery system with optimal efficacy in in vivo genome editing is based on the use of recombinant adeno-associated virus (AAV), but may cause adverse effects ( Dubey and Mostafavi, 2023 ). Therefore, lipid NPs represent an alternative ( Escalona-Rayo et al, 2023 ).…”

The Current Situation and Development Prospect of Whole-Genome Screening

“…On the other hand, the difficult is that LNPs can be exocytosed. Recently, an increase in the number of modified nanoparticles has been reported to increase the delivery efficiency for the widespread application of gene editing, which provides hope for basic research and the clinical treatment of various diseases [ 98 , 99 , 100 , 101 , 102 , 103 , 104 , 105 , 106 , 107 , 108 , 109 , 110 ].…”