Diseases
Diseases in Gene-Editing Clinical Trials
Conditions where gene editing is being tested in people, as distinct from being discussed.
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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.
| # | Voce | Categoria | Stato |
|---|---|---|---|
| 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 |
Cosa questa lista non ti diceInclusion means research is happening, not that a treatment works or is available. Each disease page states its evidence level.