Published 3 months ago

10 patients "functionally cured" of hereditary swelling disease by new CRISPR gene therapy

Gene therapy using Crispr-Cas9 has shown promising results in treating hereditary angioedema, a genetic disorder causing severe swelling attacks.
The therapy targets the KLKB1 gene, reducing levels of a protein called plasma prekallikrein and preventing swelling attacks.
Patients treated with a single dose of the therapy have experienced reduced symptoms and improved quality of life, providing hope for a permanent cure.

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BBC News

Angiodema: Gene therapy treats painful hereditary disorder

A single dose of gene therapy was enough to stop the painful swelling attacks caused by angiodema.

3 months ago·United Kingdom

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The Guardian

Gene therapy hailed as ‘medical magic wand’ for hereditary swelling disorder

Single-dose treatment transformed lives of patients with potentially deadly condition in first human trialA groundbreaking gene therapy has been hailed as a “medical magic wand” after the treatment transformed the lives of patients with a hereditary disorder that causes painful and potentially fatal swelling.Patients who took part in the first human trial of the therapy experienced a dramatic improvement in their symptoms, and many were able to …

3 months ago·London, United Kingdom

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The Independent

Gene-editing offers hope for people with potentially fatal hereditary disorder

Hereditary angioedema is characterised by severe, painful and unpredictable swelling attacks, which can affect breathing.

3 months ago·London, United Kingdom

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NZ Herald

'I'm effectively cured': Gene-editing trial proves promising for treating rare disorder

Being “a guinea pig” in a world trial of a breakthrough gene-editing treatment was a great decision for a Northland patient suffering from a rare disorder....

3 months ago·Auckland, New Zealand

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