Per the National Institute of Health, “One of the most promising areas of research in recent years has been gene editing, including CRISPR/Cas9, for fixing misspellings in genes to treat or even cure many conditions.” In this piece we provide a clinical trial update for transthyretin (TTR) amyloidosis using this technology.
CRISPR/Cas9 – Editing the Code of Life
The scientific world is abuzz … a Nobel Prize-winning technology called CRISPR/Cas9 can now edit our DNA. This programmable gene-editing technology, which is efficient, precise, and scalable, has inspired a gold rush of countless applications in medicine, agriculture and basic science. In this blog we invite you to read more about this ground-breaking advance and how, with one course of treatment, it could potentially cure hereditary transthyretin (hATTR / ATTRv) amyloidosis.