Researchers
Adriano Montano, Alexandra Lee and Catalina Sosa.
Summary
Enzyme Replacement Therapy can be an effective treatment for Mucopolysaccharidosis IVA (Morquio A disease) by reducing the accumulation of glycosaminoglycans. But immune response against the GALNS protein limits it effectiveness. In silico analysis of the cDNA sequences and in vitro testing of re-engineered protein resulted in three mutated GALNS proteins with reduced immunogenicity that exhibited 80 percent or more enzyme activity compared to the wild-type GALNS protein in vitro. Ultimately, a less immunogenic GALNS could remove the two to three year period in which the patient develops tolerance to the protein and eliminate the need for immunosuppressive protocols.
Intellectual Property Status
- U.S. patent pending