Diseases

Diseases in Gene-Editing Clinical Trials

Conditions where gene editing is being tested in people, as distinct from being discussed.

이 목록이 만들어지는 방식

These are conditions where editing has reached actual patients in registered trials. It is a much longer list than the approved one and a much shorter list than the diseases people expect CRISPR to solve.

#	항목	카테고리	상태
1	Sickle Cell Disease An inherited blood disorder in which a single DNA letter change makes red blood cells…	Blood disorders	Approved
2	Beta Thalassemia An inherited disorder in which the body makes too little beta-globin, leaving many…	Blood disorders	Approved
3	ATTR Amyloidosis A disease in which a misfolded liver protein accumulates in the heart and nerves — and…	Metabolic disease	Phase III
4	Hereditary Angioedema A rare disorder causing sudden, unpredictable and sometimes life-threatening swelling…	Rare genetic disease	Phase III
5	High Cholesterol and Familial Hypercholesterolemia The most common cause of premature heart disease, and the first attempt to treat an…	Cardiovascular disease	Phase II
6	Cancer Not one disease but hundreds — and the place where gene editing is used to engineer…	Cancer	Phase II
7	Leukemia and Lymphoma Blood cancers, where engineered cell therapy has worked best — and where the first…	Cancer	Phase II
8	HIV A virus that hides inside the genome of the cells it infects — which is why editing has…	Infectious disease	Phase I
9	Duchenne Muscular Dystrophy A severe muscle-wasting disease caused by mutations in the largest gene in the human…	Muscular disease	Preclinical
10	Cystic Fibrosis A disease of thick mucus in the lungs and gut where highly effective drugs already exist…	Rare genetic disease	Preclinical
11	Hemophilia An inherited bleeding disorder in which gene addition reached approval first, leaving…	Blood disorders	Preclinical
12	Inherited Blindness Inherited retinal disease was where in vivo CRISPR was first injected directly into a…	Eye disease	Phase I/II
13	Alpha-1 Antitrypsin Deficiency A single-letter mutation that damages the lungs by absence and the liver by accumulation…	Rare genetic disease	Phase III
14	Chronic Granulomatous Disease A rare immune disorder in which white blood cells cannot kill the bacteria they engulf —…	Rare genetic disease	Phase I/II
15	Huntington's Disease A fatal inherited brain disease caused by a toxic protein — where the genetics are…	Neurological disease	Preclinical
16	Parkinson's Disease A common neurodegenerative disease that is mostly not genetic — which makes it a poor fit…	Neurological disease	Discovery
17	Alzheimer's Disease The most common cause of dementia, where a well-known risk gene exists but editing…	Neurological disease	Discovery
18	Diabetes Where editing is being used not to correct a gene but to hide transplanted…	Metabolic disease	Phase I
19	Retinitis Pigmentosa A group of inherited retinal degenerations with over 80 causative genes — where the…	Eye disease	Preclinical

이 목록이 알려주지 않는 것Inclusion means research is happening, not that a treatment works or is available. Each disease page states its evidence level.

Written by The CRISPR Atlas editorial team Last updated Aug 25, 2026