CRISPR Decoded
Genes
The specific stretches of DNA that editing programmes aim at, and what each one does in the body.
19 genes in CRISPR Decoded.
19 shown
| Symbol | Full name | Chromosome | What it does | ★ |
|---|---|---|---|---|
| HBB | Haemoglobin subunit beta |
|
Encodes the beta chain of adult haemoglobin; a single-letter change in it causes sickle cell disease, and… | |
| BCL11A | BAF chromatin remodelling complex subunit BCL11A |
|
The switch that turns off fetal haemoglobin after birth, and the actual target of the world's first approved… | |
| PCSK9 | Proprotein convertase subtilisin/kexin type 9 |
|
A liver gene that controls how much LDL cholesterol stays in the blood, and the target of the first attempt… | |
| TTR | Transthyretin |
|
Encodes a liver protein that carries thyroid hormone and vitamin A, and which, when it misfolds, forms the… | |
| CFTR | Cystic fibrosis transmembrane conductance regulator |
|
Encodes a chloride channel in cell membranes; when it fails, secretions across the body become abnormally… | |
| DMD | Dystrophin |
|
The largest gene in the human genome, encoding dystrophin, the shock absorber that keeps muscle fibres intact… | |
| CCR5 | C-C chemokine receptor type 5 |
|
An immune cell surface receptor that HIV uses as a doorway, and the gene at the centre of the field's worst… | |
| SERPINA1 | Serpin family A member 1 |
|
Encodes alpha-1 antitrypsin, the protein that protects lung tissue from being digested by the body's own… | |
| KLKB1 | Kallikrein B1 |
|
Encodes prekallikrein, an upstream component of the pathway that produces the swelling of hereditary… | |
| HTT | Huntingtin |
|
Encodes huntingtin; an expanded CAG repeat in it causes Huntington's disease through a toxic gain of function. | |
| NCF1 | Neutrophil cytosolic factor 1 |
|
Encodes p47phox, part of the enzyme complex white blood cells use to kill bacteria, and the first gene… | |
| BRCA1 | BRCA1 DNA repair associated |
|
A DNA repair gene whose loss substantially raises the risk of breast and ovarian cancer, important to gene… | |
| BRCA2 | BRCA2 DNA repair associated |
|
The second major hereditary breast and ovarian cancer gene, also central to repairing double-strand DNA… | |
| LDLR | Low-density lipoprotein receptor |
|
The receptor liver cells use to clear LDL cholesterol from the blood; faults in it are the commonest cause of… | |
| CEP290 | Centrosomal protein 290 |
|
A large gene required for the function of the connecting cilium in photoreceptors; one deep intronic variant… | |
| APOE | Apolipoprotein E |
|
A lipid-transport gene whose ε4 variant is the strongest common genetic risk factor for late-onset… | |
| TRAC | T cell receptor alpha constant |
|
The T-cell receptor gene that must be removed to make donor immune cells safe to give to an unrelated patient. | |
| B2M | Beta-2 microglobulin |
|
A required component of MHC class I; removing it hides transplanted cells from the recipient's killer T cells. | |
| PDCD1 | Programmed cell death 1 |
|
Encodes PD-1, the brake tumours press to switch off attacking T cells, removed by editing so engineered cells… |
Gene symbols follow HGNC nomenclature; cytogenetic locations are as published by NCBI Gene and Ensembl.
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