Cell and gene therapy is the use of gene therapy vector to transduce exogenous therapeutic genes to cells, and then through the transcription and translation of exogenous genes to change the expression of original genes to treat disease. As a new generation of precision therapy after small molecule and macromolecule targeted therapy, cell and gene therapy provides new treatment concepts and means for tumors, rare diseases, chronic diseases and other refractory diseases. Viral vectors such as adenovirus, adeno-associated viruses, and retroviruses are efficient delivery systems of genetic material that can be used for cell versus gene therapy and vaccine production. Lentiviruses can be used in novel cellular immunotherapy (genetically modified cell therapy), such as CAR-T cell therapy. These innovative methods will become an important part of the new generation of serious disease therapy.
CGT techniqueflow Taking CAR-T production as an example, the specific process involves the production of plasmids, viruses, cells:
(1) Plasmid production generally takes 2 weeks. As a gene carrier, it is often used as a basic platform for gene expression, gene editing tools (such as CRISPR-Cas series) and virus production in CGT;
(2) Virus production generally takes 2-3 weeks. As a commonly used gene delivery tool for CGT, it can be used either as the final product of gene therapy, for direct in vivo therapy or as an intermediate vehicle to deliver the target gene to cells cultured in vitro. (3) Cell expansion and culture generally take 2 weeks. As the final product of cell therapy, it is usually obtained from human cells after genetic modification and amplification.
Tool cell culture with viruses mainly includes adherent culture and suspension culture. Adherent culture is the traditional mainstream method, but suspension culture can achieve higher density cell growth, that is, the cost of virus production is lower, and it is gradually becoming the mainstream. Compared to adherent cells, suspension culture allows more convenient amplification of production scale without the need for cumbersome cell isolation steps.CEKG existing pendulum bioreactors, glass bioreactors and stainless steel bioreactors can all be used to promote the growth of suspended cells.
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