SCIENCE

NellOne Therapeutics is developing a technology based on a compound that, in turn, is based on the naturally occurring cell-signaling Nell1 protein.

Analysis shows that the protein is essential for the proper formation of the heart's extracellular matrix (ECM), which constitutes the main structural components of heart muscle and imparts strength, flexibility and correct contraction. ECM is a complex mix of collagens and proteoglycans.

Changes in the cardiac ECM play critical roles in the calcification of heart valves and in the degree of heart muscle damage incurred after a heart attack.

Studies in Nell1-deficient mice clearly demonstrated the importance of Nell1 protein in cell maturation of certain cells and tissues. It promotes cell maturation in the cardiovascular system by controlling the pathway of downstream genes that are needed for the proper deposition and composition of the ECM.

High-through put gene expression analyses completed the picture by revealing the Nell1 pathway members and the possible mechanisms by which the Nell1 protein exerts its influence on cell growth and differentiation.