Table 1.

Definition of genome-editing products

• In vivo genome-editing products (gene therapy products that use at least one genome-editing technology and that are administered directly in the body)

➢ Gene therapy vector products for genome editing (gene therapy products consisting of a viral or plasmid vector that expresses desired proteins, that is, nucleases used for genome editing) and gRNAs for genome editing

➢ mRNA products for genome editing (mRNA that expresses desired proteins for genome editing)

➢ Protein products for genome editing (desired proteins or protein/gRNA complexes used for genome editing)

• Ex vivo genome-editing products (human cell-based products genetically modified by a genome-editing tool)

gRNA, guide RNA; mRNA, messenger RNA.