In a new study, patients with the genetic disorder hereditary angioedema (HAE) showed significant improvement in symptoms after treatment with CRISPR-Cas9, a genome editing tool.
HAE is a rare, potentially life-threatening condition that causes episodes of swelling in the face, hands, feet, genitals, abdomen, and larynx. Current treatments are limited and often ineffective, so there is a great need for new therapeutic options.
The new study, published in the journal Nature Medicine, used CRISPR-Cas9 to target a faulty gene that causes HAE. This approach was able to successfully decrease the levels of the faulty protein and reduce symptoms in HAE patients.
While this is a small study and more research is needed, the results are promising and suggest that CRISPR-Cas9 could be a potential treatment for HAE. This approach could also be used to treat other genetic disorders.
A new CRISPR-based treatment for hereditary angioedema (HAE) shows promise in reducing the severity and duration of attacks, according to a study published in the New England Journal of Medicine.
HAE is a rare, autosomal dominant disorder characterized by episodes of swelling in various parts of the body. Attacks can be triggered by emotions, physical activity, or infections and can be very painful. There is currently no cure for HAE, and treatment focuses on managing symptoms and preventing attacks.
The new CRISPR-based treatment, which is still in the early stages of development, uses a livestock virus to deliver a therapeutic gene to the patient’s cells. The treatment was tested in a small group of HAE patients, and the results were promising.
Patients who received the treatment had a significant reduction in the number and severity of attacks. In addition, the duration of attacks was shorter and the time between attacks was longer.
The CRISPR-based treatment is still in the early stages of development and more research is needed to confirm its safety and efficacy. However, the results of this study are promising and offer a new hope for patients with HAE.
