A new type of therapy that 'edits' a gene in patients with a rare heart condition has been shown to be safe and effective, according to research from UCL and the Royal Free Hospital.
University College LondonNov 20 2024 A new type of therapy that 'edits' a gene in patients with a rare heart condition has been shown to be safe and effective, according to research from UCL and the Royal Free Hospital .
The new therapy, which uses the Nobel Prize-winning CRISPR-Cas9 gene editing technology, works by inactivating a gene in the liver that is responsible for production of transthyretin, resulting in the production of only negligible quantities of transthyretin. This is really promising news for people with this awful condition. The hope is that, in patients diagnosed in the early stage of the disease, this treatment will offer the prospect of preserving a good quality of life without the need for ongoing therapy."
Heart Amyloidosis Cas9 Clinical Trial CRISPR Heart Failure Hospital Medicine Protein Research Technology
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