New Cell-specific ERT Shows Promise in Mouse Study

New Cell-specific ERT Shows Promise in Mouse Study

307482

New Cell-specific ERT Shows Promise in Mouse Study

A new enzyme replacement therapy (ERT) targeting specific cell types boosts the delivery of acid alpha-glucosidase (GAA) — the enzyme missing or defective in Pompe disease patients — to muscle and heart cells, a study shows. Compared to standard ERT, the targeted approach by Regeneron Pharmaceuticals normalized the levels of glycogen — the complex sugar molecule that builds up in Pompe patients — in a mouse model of the disease, particularly in skeletal muscles. Also, the…

You must be logged in to read/download the full post.