Mucolipidosis IV Foundation

Welcome to the Mucolipidosis IV Foundation website at ml4.org. We hope you find our web design easy to use and the information helpful.

What the ML4 Foundation has already accomplished

• The gene responsible for ML4 was discovered in 2000.
• A carrier test for ML4 was created in 2003.
• A mouse model for ML4 was developed in 2007.
• A fruit fly model for ML4 was developed in 2008.

Here is a link to an article (and video) about the ML4 fruit fly model.
http://www.hopkinsmedicine.org/Press_releases/2008/11_26_08.html

Current Research

• Drug testing on the ML4 mouse.
• Researching various delivery systems aimed at crossing the blood brain barrier to deliver normal cells to the brain.
• A natural history study of ML4 patients.
• Studying iron transport issues in ML4.