Genome editing

Genome editing is the deliberate alteration of DNA at targeted locations, typically by removing, adding, or changing genetic sequences. It can modify genes in cells or organisms and may be inherited when reproductive cells are edited.

CRISPR-Cas9: A genome-editing system that uses guide RNA to direct the Cas9 enzyme to a chosen DNA sequence. Its programmable targeting made genome editing faster and more accessible.
Somatic gene therapy: Treatment that changes genes in a patient’s non-reproductive cells to prevent or treat disease. Genome editing can alter disease-related genes without passing changes to offspring.
Transgenesis: The introduction of genetic material from one organism into another organism’s genome. Genome editing can change existing sequences without necessarily adding foreign DNA.
Off-target effect: An unintended change or biological effect caused when a molecular tool acts outside its intended target. Unintended edits can complicate safety assessments and clinical use.
Restriction enzyme: An enzyme that recognizes specific DNA sequences and cuts DNA at or near those sites. Restriction enzymes established the use of sequence-specific cutting in molecular biology.
Base editing: A genome-editing method that converts one DNA base into another without making a double-strand break. It can correct some point mutations without cutting both DNA strands.
Sickle cell disease: An inherited blood disorder caused by mutations in the gene encoding beta-globin. Edited blood stem cells provide a prominent clinical application of genome editing.
Gene therapy: The treatment of disease by altering, replacing, or supplementing genetic material in cells. Genome editing is one strategy within the broader field of gene therapy.
Germline editing: Genetic alteration of reproductive cells or embryos that can be inherited by descendants. Heritable edits raise distinct ethical and governance questions from treatment of somatic cells.
Recombinant DNA: DNA molecules formed by joining genetic sequences from different sources. Recombinant DNA methods enabled deliberate genetic construction before targeted genome editing.

Linked from 14 pages