CRISPR Cas-9 as a Potential Cure for Wilson's Disease by Satoshi Toya
- Satoshi Toya
- Nov 27
- 1 min read
Wilson's Disease is an inherited copper metabolism disorder that is characterized by the pathological accumulation of copper throughout the body. It is caused by a mutation in the ATP7B gene – a gene that encodes a transmembrane copper-transporting protein – which inhibits the body's ability to transport copper properly. This results in the accumulation of copper in organs such as the brain and liver, which cause ophthalmological, hepatic, neurological, and psychiatric symptoms. The primary methods of treatment are chelators and zinc salts, which lower copper levels through different mechanisms; these treatments can become expensive because they need to be used continuously throughout the patient's lifetime.
However, CRISPR – an immune system used by various bacteria and archaea to eliminate invading viruses and plasmids – can be utilized as an effective gene editor, with many possible applications, including the treatment of chronic genetic diseases. The most prominent version of CRISPR used for this purpose is the CRISPR-Cas9 system. This paper aims to achieve multiple goals: to explain Wilson's Disease, mechanisms, and symptoms; to overview CRISPR and how CRISPR-Cas9 can modify DNA in genes; and to propose how CRISPR could be used to potentially permanently cure chronic genetic diseases, such as Wilson's Disease.
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