Mucolipidosis IV Foundation
ML4 is a genetic disease causing mental retardation, retinal degeneration, and limited lifespan. The mission of the Foundation is to fund, promote, and support medical research dedicated to developing treatments and a cure for ML4.
Scientific Research
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.
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.
Randy Gold, ML4 Foundation Vice President, was named the 2011 Jewish Community Hero sponsored by the Jewish Federations® of North America for his efforts around promoting awareness of the 19 known preventable Jewish Genetic Diseases.
