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Overview and Vectors For Gene Therapy On January 25, 2022, Nature published an article listing seven technologies worthy of attention this year. Targeted genetic therapies was on the list. The remaining six technologies are: Fully finished genomes, Protein structure solutions, Quantum simulation, Precise genome manipulation, Spatial multi-omics), CRISPR-based diagnostics.
An overview of gene therapy Gene therapy refers to the delivery of exogenous genes with normal functions to target cells in the human body with a certain carrier, and the purpose of treating diseases is achieved by correcting the defective genes. If chemotherapy is a "symptom cure", then gene therapy is a "cure for the root cause". With the gradual emergence of gene therapy in clinical applications, it has become one of the most rapidly developing directions in the field of biomedicine in recent years.
Most gene therapy is to obtain modified functional cells locally or in vitro, and then transplant them into the patient. However, this method is limited in drug delivery and cannot precisely deliver the drug to the target tissue. Of course, except for the liver, because the liver has the function of filtering blood, in addition to intravenous infusion, even subcutaneous injection can achieve specific targeted delivery to the liver. For gene drug delivery in extrahepatic tissues, major pharmaceutical companies are making steady progress to seize key technologies. Drug carrier is the key technology of gene therapy, and commonly used carriers can be divided into viral vectors and non-viral vectors.
Gene Therapy Vectors Depending on the source and nature of the vector, gene therapy vectors can be divided into two categories: viral vectors and non-viral vectors. Viral vectors mainly include