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.

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Blood disorders Sickle Cell DiseaseAn inherited blood disorder in which a single DNA letter change makes red blood cells stiffen into crescents… Blood disorders Beta ThalassemiaAn inherited disorder in which the body makes too little beta-globin, leaving many patients dependent on… Metabolic disease ATTR AmyloidosisA disease in which a misfolded liver protein accumulates in the heart and nerves — and the setting for the… Rare genetic disease Hereditary AngioedemaA rare disorder causing sudden, unpredictable and sometimes life-threatening swelling attacks — and the… Cardiovascular disease High Cholesterol and Familial HypercholesterolemiaThe most common cause of premature heart disease, and the first attempt to treat an ordinary chronic… Cancer CancerNot one disease but hundreds — and the place where gene editing is used to engineer immune cells rather than… Cancer Leukemia and LymphomaBlood cancers, where engineered cell therapy has worked best — and where the first gene-edited cells were… Infectious disease HIVA virus that hides inside the genome of the cells it infects — which is why editing has been proposed both to… Muscular disease Duchenne Muscular DystrophyA severe muscle-wasting disease caused by mutations in the largest gene in the human genome — and the… Rare genetic disease Cystic FibrosisA disease of thick mucus in the lungs and gut where highly effective drugs already exist for most patients —… Blood disorders HemophiliaAn inherited bleeding disorder in which gene addition reached approval first, leaving editing to solve the… Eye disease Inherited BlindnessInherited retinal disease was where in vivo CRISPR was first injected directly into a human organ — the eye… Rare genetic disease Alpha-1 Antitrypsin DeficiencyA single-letter mutation that damages the lungs by absence and the liver by accumulation — and the first… Rare genetic disease Chronic Granulomatous DiseaseA rare immune disorder in which white blood cells cannot kill the bacteria they engulf — and the first… Neurological disease Huntington's DiseaseA fatal inherited brain disease caused by a toxic protein — where the genetics are unusually clear and the… Neurological disease Parkinson's DiseaseA common neurodegenerative disease that is mostly not genetic — which makes it a poor fit for gene editing… Neurological disease Alzheimer's DiseaseThe most common cause of dementia, where a well-known risk gene exists but editing remains firmly a… Metabolic disease DiabetesWhere editing is being used not to correct a gene but to hide transplanted insulin-producing cells from the… Eye disease Retinitis PigmentosaA group of inherited retinal degenerations with over 80 causative genes — where the obstacle is not delivery…