O CRISPR Atlas

Diseases

Conditions where gene editing is approved, in trials, or still only a laboratory idea — with that difference stated on every page.

19 diseases in the Atlas. Every disease page states whether gene editing for that condition is an approved treatment, in clinical trials, in preclinical work, or still only a laboratory idea — that distinction is the most important thing on the page.

19 exibidos

Blood disorders 3

Sickle Cell Disease HBB · An inherited blood disorder in which a single DNA letter… Beta Thalassemia HBB · An inherited disorder in which the body makes too little… Hemophilia An inherited bleeding disorder in which gene addition…

Metabolic disease 2

ATTR Amyloidosis TTR · A disease in which a misfolded liver protein accumulates… Diabetes Where editing is being used not to correct a gene but to…

Rare genetic disease 4

Hereditary Angioedema KLKB1 · A rare disorder causing sudden, unpredictable and… Cystic Fibrosis CFTR · A disease of thick mucus in the lungs and gut where… Alpha-1 Antitrypsin Deficiency SERPINA1 · A single-letter mutation that damages the lungs by… Chronic Granulomatous Disease NCF1 · A rare immune disorder in which white blood cells cannot…

Cardiovascular disease 1

High Cholesterol and Familial Hypercholesterolemia PCSK9 · The most common cause of premature heart disease, and the…

Cancer 2

Cancer Not one disease but hundreds — and the place where gene… Leukemia and Lymphoma Blood cancers, where engineered cell therapy has worked…

Infectious disease 1

HIV CCR5 · A virus that hides inside the genome of the cells it…

Muscular disease 1

Duchenne Muscular Dystrophy DMD · A severe muscle-wasting disease caused by mutations in…

Eye disease 2

Inherited Blindness CEP290 · Inherited retinal disease was where in vivo CRISPR was… Retinitis Pigmentosa A group of inherited retinal degenerations with over 80…

Neurological disease 3

Huntington's Disease HTT · A fatal inherited brain disease caused by a toxic protein… Parkinson's Disease A common neurodegenerative disease that is mostly not… Alzheimer's Disease APOE · The most common cause of dementia, where a well-known…