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Gene therapy is an experimental technique that uses proteins to treat or prevent disease. It targets the faulty genes responsible for genetic diseases.
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European Medicines Agency (EMA) defines 'Gene Therapy Medicinal Products (GTMPs)' as: "generally consist of a vector or delivery formulation/system containing a genetic construct engineered to express a specific transgene ('therapeutic sequence') for the regulation, repair, replacement, addition or deletion of a genetic sequence. The active substance is the nucleic acid sequence(s), or genetically modified microorganism(s), virus(es) or cells."
EMA defines that a gene therapy medicinal product is a biological medicinal product which fulfills the following two characteristics: (a) it contains an active substance which contains or consists of a recombinant nucleic acid used in or administered to human beings with a view to regulating, repairing, replacing, adding or deleting a genetic sequence; (b) its therapeutic, prophylactic or diagnostic effect relates directly to the recombinant nucleic acid sequence it contains, or to the product of genetic expression of this sequence.
Gene therapy medicinal products shall include vaccines against infectious diseases.
During gene editing by CRISPR/Cas9, the genes encoding the Cas9 protein and sgRNA are introduced into a cell where a targeted genomic change is planned. The sgRNA has a region complementary to the chosen genomic target sequence (purple); this region can be engineered to include any desired sequence. A complex consisting of the CRISPR sgRNA and the Cas9 protein forms within the cell & binds to the chosen target site in the DNA.
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