Gene therapy refers to a medical intervention that modifies or replaces defective genes within an individual's cells to treat or prevent diseases. ExCell Bio has developed series of products，services to support your research.
Preparation of Target Gene
To determine a target gene for therapeutic purposes, if a disease is identified to result from abnormalities in a specific gene, its wild-type counterpart can be utilized in gene therapy. For instance, the wild-type ADA gene is employed to treat ADA deficiency.
Plasmid Production
Large-scale generation of circular DNA plasmids (typically in E. coli via fermentation) for applications like gene therapy vector packaging, mRNA vaccine templates, or recombinant protein expression.
Construction of Vector
Vectors are responsible for delivering exogenous genes into target cells, categorized into viral vectors and non-viral vectors. Viruses currently utilized as vectors include retroviruses, adenoviruses, adeno-associated viruses (AAV), herpesviruses, and hepatitis viruses.
Cell Transfection and expansion
Genes can be introduced into target cells via viral infection, physical methods (e.g., electroporation, gene gun), or chemical methods (e.g., lipid nanoparticles, calcium phosphate) . However, current transfection efficiency remains suboptimal (below 100%) due to factors such as cell type variability, vector size, and nucleic acid purity.The cultivation of HEK293 cells is pivotal for the production of final therapeutic products due to their high transfection efficiency and capacity for recombinant protein/viral vector expression.
Virus Purification and quality control
A critical downstream bioprocessing step to isolate and concentrate intact viral particles (e.g., adenovirus, lentivirus, AAV) from crude lysates or cell culture supernatants while removing contaminants like host cell proteins, DNA, and empty capsids.
