Introduction to gene delivery vectors for gene drugs

Gene drugs must first solve the problem of target gene delivery vector systems. The vector must be able to efficiently and safely deliver the target gene to the nucleus of specific diseased tissues in order to express the function of the target gene. The virus vector delivery system is the most widely used system, accounting for about 90%.

The world's first approved tumor gene therapy drug, Gendicine, uses an adenovirus vector delivery system. The large-scale industrialization technology of Gendice has been tested for over 20 years and its safety has been globally recognized. The most famous academic journal in the field of genetics, "Human Gene Therapy," has published articles that highly recognize Genidine and its industrialization system.

Among the globally approved gene drugs, adenovirus, adeno-associated virus, and retrovirus are mainly used as vectors. The Ebola gene vaccine in China uses adenovirus vector, while the Ebola gene vaccine in the United States uses herpes virus vector.

Adenovirus vector is a high molecular weight vector that can carry large target genes or multiple target genes. If it is compared to a carrier rocket, adenovirus can be understood as a high thrust carrier rocket with broad application prospects; It is particularly important that adenovirus does not integrate into the human genome and does not pose potential risks to the human body.

Adeno-associated virus (AAV) is a small molecular weight vector that can only carry small target genes; Adeno associated viruses pose a potential risk of integration into the human genome, which is why many gene drugs used to treat genetic diseases use this virus as a target gene delivery vector. We know that if the virus is integrated into the human genome, it will cause genomic instability and pose potential risks to the human body, which have been mentioned in many authoritative academic papers.