Introduction
Recently, the importance of gene therapy in disease treatment has been highly noted. However, conventional therapeutic nucleic acid carriers are often produced by two-step synthesis-packing procedure, leading to relative drawbacks in stability and ease of storage of the products. Moreover, traditional processes typically only allow for the delivery of a single gene, limiting their applications. Here, we develop a technology to create a stable nucleic acid delivery carrier with multiple targets, functionalities, and customization capabilities, offering personalized treatments tailored to the molecular characteristics of diseases.
This technology employs a biological process to produce stable nucleic acid carriers, utilizing a gene similar to bacteriophages to establish self-assembling virus-like nucleic acid carriers. The carrier's framework consists of three unique functional domains, enabling the transport of functional nucleic acids such as siRNA, miRNA, fluorescence tracking aptamers, or targeting aptamers. This nucleic acid carrier exhibits modular multi-functional design, automatic encapsulation of nucleic acids, and stable biological processes, providing several advantages that effectively and precisely regulate the types of nucleic acids carried and enhance their stability. Safety verification, efficacy validation, and preliminary pilot production verification have all demonstrated the substantial potential of this carrier for clinical applications. In the future, it holds the opportunity to significantly improve the efficiency of gene therapy, benefiting patients extensively.
Features / strengths
Recently, the importance of gene therapy in disease treatment has been highly noted. However, conventional therapeutic nucleic acid carriers are often produced by two-step synthesis-packing procedure, leading to relative drawbacks in stability and ease of storage of the products. Moreover, traditional processes typically only allow for the delivery of a single gene, limiting their applications. Here, we develop a technology to create a stable nucleic acid delivery carrier with multiple targets, functionalities, and customization capabilities, offering personalized treatments tailored to the molecular characteristics of diseases.
This technology employs a biological process to produce stable nucleic acid carriers, utilizing a gene similar to bacteriophages to establish self-assembling virus-like nucleic acid carriers. The carrier's framework consists of three unique functional domains, enabling the transport of functional nucleic acids such as siRNA, miRNA, fluorescence tracking aptamers, or targeting aptamers. This nucleic acid carrier exhibits modular multi-functional design, automatic encapsulation of nucleic acids, and stable biological processes, providing several advantages that effectively and precisely regulate the types of nucleic acids carried and enhance their stability. Safety verification, efficacy validation, and preliminary pilot production verification have all demonstrated the substantial potential of this carrier for clinical applications. In the future, it holds the opportunity to significantly improve the efficiency of gene therapy, benefiting patients extensively.
Specification in detail