experimental therapeutic technique that manipulates genes to treat or prevent disease. The procedure assumes the transplantation of working genes into cells in place of missing or defective ones, and allows rescue of normal functions or the addition of new functions to cells to combat diseases.
DNAi is inserted, deleted or replaced using "molecular scissors" - engineered nucleases creating site-specific double-strand breaks at desired locations in the genome. These breaks are further edited and repaired through nonhomologous end-joining or homologous recombination.
There are currently four families of engineered nucleases being used: meganucleases, zinc finger nucleases, transcription activator-like effector-based nucleases, and the CRISPR-Cas system.
DNAi is inserted, deleted or replaced using "molecular scissors" - engineered nucleases creating site-specific double-strand breaks at desired locations in the genome. These breaks are further edited and repaired through nonhomologous end-joining or homologous recombination.
There are currently four families of engineered nucleases being used: meganucleases, zinc finger nucleases, transcription activator-like effector-based nucleases, and the CRISPR-Cas system.
