Genome editing is a technique by which DNA is inserted, replaced, removed, or modified at particular locations in the human genome for therapeutic benefit in order to treat cancer, rare inherited disorders, HIV, or other diseases. Several approaches rely on the use of “molecular scissors,” often an engineered nuclease, to make precise cuts in the patient’s DNA at a specific location in the genome. The breaks are then repaired to create the desired edit and result in a corrected gene.
Genome editing nucleases that are currently used in genome editing include: meganucleases, zinc finger nucleases (ZFNs), transcription activator-like effector-based nucleases (TALEN), and nucleases such as Cas9 and Cas 12a that derive from the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR/Cas). Alternatively, genome editing can also be performed by homologous recombination of adeno-associated virus (AAV)-derived sequences into the patient’s DNA.
Examples of ARM members involved in genome editing include: