Genes
Most Studied Genes in Gene Editing
The genes that editing programmes actually aim at, and why each was chosen.
How this list is built
Editing programmes cluster around a small number of genes, and the reasons are consistent: the gene is in the liver where delivery works, or switching it off is known to be safe because people are born without it, or the disease is caused by too much of something rather than too little.
| # | Entry | Category | Status |
|---|---|---|---|
| 1 | HBB Encodes the beta chain of adult haemoglobin; a single-letter change in it causes sickle… | Blood | |
| 2 | BCL11A The switch that turns off fetal haemoglobin after birth — and the actual target of the… | Blood | |
| 3 | PCSK9 A liver gene that controls how much LDL cholesterol stays in the blood — and the target… | Cardiovascular | |
| 4 | TTR Encodes a liver protein that carries thyroid hormone and vitamin A — and which, when it… | Metabolic | |
| 5 | CFTR Encodes a chloride channel in cell membranes; when it fails, secretions across the body… | Respiratory | |
| 6 | DMD The largest gene in the human genome, encoding dystrophin — the shock absorber that keeps… | Muscle | |
| 7 | CCR5 An immune cell surface receptor that HIV uses as a doorway — and the gene at the centre… | Immune | |
| 8 | SERPINA1 Encodes alpha-1 antitrypsin, the protein that protects lung tissue from being digested by… | Metabolic | |
| 9 | KLKB1 Encodes prekallikrein, an upstream component of the pathway that produces the swelling of… | Metabolic | |
| 10 | HTT Encodes huntingtin; an expanded CAG repeat in it causes Huntington's disease through a… | Neurological | |
| 11 | NCF1 Encodes p47phox, part of the enzyme complex white blood cells use to kill bacteria — and… | Immune | |
| 12 | BRCA1 A DNA repair gene whose loss substantially raises the risk of breast and ovarian cancer —… | Cancer | |
| 13 | BRCA2 The second major hereditary breast and ovarian cancer gene, also central to repairing… | Cancer | |
| 14 | LDLR The receptor liver cells use to clear LDL cholesterol from the blood; faults in it are… | Cardiovascular | |
| 15 | CEP290 A large gene required for the function of the connecting cilium in photoreceptors; one… | Eye | |
| 16 | APOE A lipid-transport gene whose ε4 variant is the strongest common genetic risk factor for… | Neurological | |
| 17 | TRAC The T-cell receptor gene that must be removed to make donor immune cells safe to give to… | Immune | |
| 18 | B2M A required component of MHC class I; removing it hides transplanted cells from the… | Immune | |
| 19 | PDCD1 Encodes PD-1, the brake tumours press to switch off attacking T cells — removed by… | Immune |
What this list does not tell youOrdered by prominence in current editing programmes, which is an editorial judgement rather than a measurement.