Table 1.
Glossary of terms
AAV (adeno-associated virus) | A viral vector system used for gene delivery. |
Chimera | A single organism composed of cells from different zygotes. |
Germ line therapy | Insertion of DNA into germ line cells (egg or sperm) so that the offspring will have the inserted gene. |
gRNA | Guide RNA. |
Hematopoietic stem cells | Unspecialized precursor cells that will develop into mature blood cells. |
Pluripotent stem cells | Stem cells that can become all cell types found in an implanted embryo, fetus, or developed organism (excluding trophoblast and placenta). |
Recombinant DNA | A novel DNA sequence formed by the joining, usually in vitro, of two non-homologous DNA molecules. |
Retroviral vector | A disabled RNA virus in which the viral genes have been replaced with engineered sequences. |
RuvC | An endonuclease domain named for an E. coli protein involved in DNA repair. |
sgRNA | Single guide RNA. |
Stem cells | Cells with the ability to divide for indefinite periods in culture and to give rise to specialized cells. |
tracrRNA, trRNA | Trans-activating crRNA. |