The definitive guide to gene editing.
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CRISPR Decoded

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 CRISPR Decoded. 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 shown

Blood disorders 3

Blood smear from a patient with sickle cell anemia displaying sickle cells, polychromasia, and target cells Sickle Cell Disease HBB · An inherited blood disorder in which a single DNA letter… A giemsa stained blood smear from a person with beta thalassemia Beta Thalassemia HBB · An inherited disorder in which the body makes too little… A chain of clotting proteins with one link missing Hemophilia An inherited bleeding disorder in which gene addition…

Metabolic disease 2

Fibrous amyloid deposits wrapped around heart muscle ATTR Amyloidosis TTR · A disease in which a misfolded liver protein accumulates… A pancreatic islet cell releasing insulin granules Diabetes Where editing is being used not to correct a gene but to…

Rare genetic disease 4

Swollen right hand in a female patient during a hereditary angioedema attack. Hereditary angioedema Hereditary Angioedema KLKB1 · A rare disorder causing sudden, unpredictable and… A cross-section of an airway clogged with thick mucus Cystic Fibrosis CFTR · A disease of thick mucus in the lungs and gut where… Photomicrograph of a liver biopsy from a patient with alpha-1 antitrypsin deficiency. The PAS with diastase stain shows the diastase-resistant pink globules that are characteristic 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

A liver and an artery cross-section with lipoprotein particles High Cholesterol and Familial Hypercholesterolemia PCSK9 · The most common cause of premature heart disease, and the…

Cancer 2

Micrograph of a nodular basal cell cancer with a cleft. H&E stain. Such clefts are caused by shrinkage of mucin during tissue fixation and staining. Robert S Bader. Which histologi Cancer Not one disease but hundreds, and the place where gene… Blood smear showing acute lymphatic leukaemia (Jenner's stain). Wellcome Images Keywords: Clinical Medicine; Goodall; Haematology; Guilland; Leukaemia Leukemia and Lymphoma Blood cancers, where engineered cell therapy has worked…

Infectious disease 1

This scanning electron micrograph revealed the presence of the human immunodeficiency virus (HIV-1), (spherical in appearance), which had been co-cultivated with human lymphocytes. HIV CCR5 · A virus that hides inside the genome of the cells it…

Muscular disease 1

Histopathology of gastrocnemius muscle from patient who died of pseudohypertrophic muscular dystrophy, Duchenne type. Cross section of muscle shows extensive replacement of muscle Duchenne Muscular Dystrophy DMD · A severe muscle-wasting disease caused by mutations in…

Eye disease 2

Rows of retinal photoreceptor cells, some degenerating 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

Coronal FSPGR through the brain at the level of the caudate nuclei demonstrating marked reduced volume in keeping with the patient's known diagnosis of Huntington's Disease. Image Huntington's Disease HTT · A fatal inherited brain disease caused by a toxic… A network of neurons with a few glowing brightly Parkinson's Disease A common neurodegenerative disease that is mostly not… Tangled, degenerating neurons with amber deposits Alzheimer's Disease APOE · The most common cause of dementia, where a well-known…
Reading these pages A disease appearing here does not mean gene editing can treat it. Most entries describe research, and most research programmes never become medicines. Each page labels its evidence level at the top.