Diseases
Diseases With an Approved Gene-Editing Treatment
The conditions where gene editing has crossed from research into approved medicine.
How this list is built
Two conditions, both blood disorders, both treatable by the same intervention. That is the entire list — and understanding why it is so short tells you more about this field than any success story.
| # | Entry | Category | Status |
|---|---|---|---|
| 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 |
What this list does not tell youAn approved treatment existing does not mean it is available, affordable or suitable for any particular person.