CRISPR milestone pushes gene editing toward its promise - Axios

The gene editing system CRISPR-Cas9 can be injected into the blood and directed to the liver to treat patients with a rare condition, according to a recent study.

Why it matters: The ability to edit genes directly in a patient's body expands the list of possible diseases and conditions researchers can try to target with CRISPR-based therapies.

Driving the news: Preliminary but promising results from a small trial in six people indicate a one-time CRISPR-Cas9 treatment led to a decrease in the misfolded protein that causes the condition transthyretin amyloidosis (ATTR).

The trial results don't yet indicate whether symptoms of the disease are alleviated.

The big picture: CRISPR therapies are being developed and studied for a range of diseases and conditions.

But delivering the gene editing system to some cells without it being degraded remains a challenge for the field.

What to watch: Leonard says in addition to continuing to study the AATR therapy in pursuit of FDA approval, Intellia is looking at whether the mRNA approach could be applied to treat sickle cell anemia and eliminate the need for bone marrow transplants.

Go deeper: CRISPR co-discoverer on the gene editor's pandemic push (Axios)

https://www.axios.com/crispr-milestone-gene-editing-2306ad31-4548-4a70-b60d-91379f7722e5.html