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The treatment uses CRISPR/Cas9 editing technology, a technique derived from bacteria that allows researchers to edit the genome of a cell by specifying regions of…
Investigators have found that intravenous infusion of a CRISPR-Cas9 based gene therapy decreases serum transthyretin (TTR) protein in patients with hereditary TTR…
Researchers from Intellia Therapeutics, Regeneron Pharmaceuticals, and clinical partners have published the first-ever clinical data that support the safety and…
People with hereditary transthyretin (ATTR) amyloidosis have a mutation in the transthyretin (TTR) gene, which means they produce an abnormal protein that gradually…
The gene editor CRISPR excels at fixing disease mutations in lab-grown cells. But using CRISPR to treat most people with genetic disorders requires clearing an…
Preliminary results from a landmark clinical trial suggest that CRISPR-Cas9 gene editing can be deployed directly into the body to treat disease.
The study is the…
