NEW YORK (GenomeWeb) – Arrowhead Research today announced that it has begun dosing in a Phase I trial of its siRNA-based alpha-1 antitrypsin (AAT) deficiency therapy ARC-AAT.

AAT deficiency is a genetic disease that leads to decreased activity of the protease inhibitor alpha-1 antitrypsin in the blood and lungs causing lung dysfunction. It can also feature the accumulation of a mutant AAT protein known as Z-AAT in liver tissue, which leads to liver injury, fibrosis, cirrhosis, and potentially liver cancer. ARC-AAT is designed to lower production of Z-AAT.

Get the full story with
GenomeWeb Premium

Only $95 for the
first 90 days*

A trial upgrade to GenomeWeb Premium gives you full site access, interest-based email alerts, access to archives, and more. Never miss another important industry story.

Try GenomeWeb Premium now.

Already a GenomeWeb Premium member? Login Now.
Or, See if your institution qualifies for premium access.

*Before your trial expires, we’ll put together a custom quote with your long-term premium options.

Not ready for premium?

Register for Free Content
You can still register for access to our free content.

The apple has traveled westward and eastward along the Silk Road, according to a new genetic analysis.

In Nature this week: GWAS data used to reposition drugs for psychiatric use, and more.

Genetic disease risk information doesn't always spur people to make healthy lifestyle changes, according to the Associated Press.

A University of California, San Diego-led team has used liquid biopsies to uncover possible treatments for patients with cancers of unknown primary.

Sep
12
Sponsored by
PerkinElmer

This webinar will cover recent advances in the use of CRISPR for generating animal models and cell lines.