Figure 1.

Gene/genome therapy options for the cure of hereditary blood disorders based on gene target size. With gene transfer WT form of the disease gene is added in the genome of the recipient cells; with gene editing the mutated gene is corrected; with BAC-mediated genome transfer the mutated gene including upstream regulatory sequences and intronic regions is substituted with homologous recombination; with chromosome transplantation the entire chromosome containing the altered gene(s) is replaced with a WT chromosome. MMCT, microcell-mediated chromosome transfer.