미니서클 DNA: 유전자 치료의 미래를 여는 것

Gene therapy, as a cutting-edge therapeutic approach, brings new hope for numerous incurable diseases. Among them, minicircle DNA (mcDNA), serving as a non-viral DNA carrier, is gradually demonstrating its unique charm.

Introduction of mcDNA

mcDNA originates from plasmid DNA (pDNA) by removing prokaryotic sequences and retaining only eukaryotic sequences, forming a smaller and safer gene delivery vector. Compared to pDNA, mcDNA significantly reduces immunogenicity, avoids the risk of antibiotic resistance gene transfer, and achieves more efficient gene expression within cells, enhancing treatment efficacy.

Yaohai Bio-Pharma has established GMP-compliant production platforms for both circular and linear plasmids, backed by mature process development and GMP production experience. Yaohai can fulfill clients' different needs, ranging from preclinical research, IND submission, and clinical trials, to commercial production, and efficiently advance the progress of projects.

Production Process of mcDNA

The production of mcDNA involves steps such as parental plasmid (PP) amplification, induction of recombination, and impurity removal. Currently, various recombinase systems are used for mcDNA production, including Phage λ integrase, Phage P1 Cre recombinase, ParA resolvase, and the PhiC31-integrase/I-SceI homing endonuclease system. However, each system faces challenges in terms of yield, purity, and cost. To increase yield, researchers are exploring strategies such as modifying gene strains and optimizing fermentation conditions.

Purification of mcDNA

The purification methods for mcDNA have evolved from backbone modification to novel chromatography techniques. Despite various purification strategies, issues such as low recovery rates and high costs remain. Recent research has achieved efficient purification of mcDNA using techniques like cadaverine-modified monolithic columns, but further reductions in cost and improvements in recovery rates are still needed.

Quantitative Analysis of mcDNA

Currently, quantitative analysis of mcDNA primarily relies on methods such as qPCR and electrophoresis, which have issues with high costs and limited accuracy. The latest research has utilized chromatography technology to achieve rapid and accurate quantitative analysis of mcDNA, providing a new means for quality control of mcDNA.

Application Prospects and Challenges of mcDNA

mcDNA exhibits broad application prospects in gene therapy, DNA vaccines, and cellular therapy. However, issues such as low yield, high cost, difficult quality control, and limited in vivo delivery efficiency still need to be addressed. In the future, researchers will focus on increasing mcDNA yield, reducing costs, optimizing quality control methods, and developing more efficient delivery systems to promote the development of mcDNA in clinical applications.

결론

As an emerging non-viral DNA carrier, mcDNA holds great potential in gene therapy and related fields. Despite facing many challenges, with the advancement of research and technology, mcDNA is expected to become the most popular non-viral DNA carrier in the future, bringing breakthroughs to the medical field.

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