Rice Univeristy and Baylor College researchers have unlocked the first structural map of a protein (leiomodin 2) in order to treat a neuromuscular disease. This study could improve and help to move forward the study of nemaline myopathy, a congenital hereditary neuromuscular disorder that causes weakness of muscle where severity vary.
Conversely, biochemists Jianpeng Ma and Qinghua Wang together with their colleagues have done and presented their study in the Proceedings of the National Academy of Sciences. In this study, protein consists of amino acids, which are chained like a large molecule. Understanding its chains that lead into functional proteins does help the researchers to develop and understand the causes of disease and the treatment that may be done through drugs.
Jianpeng Ma stated that it takes too long for the protein's growth into an actin filament. She added that mutations can be used to limit actin filament growth. With this they only need two to four actin subunits to shorten the process of crystallization.
With this, Ma and Wang became more interested in Lmod2, a protein that nucleates actin and then bring about the formation of actin filaments within the muscle cells. This then expands and contract the elongated cells allowed by the bundle of filaments that form fiber-like scaffolds. Leiomodin 2 (LMOD2) is a Protein Coding gene. It is one of the family of proteins essential to actin functions in many types of muscle cells like skeletal and cardiac muscles.
Conversely, Wang said that this kind of protein is very important in biogenesis and the actin filaments maintenance, as the filaments cannot be too short or too long.
Wang and Baylor mapped a mutation of Lmod3, another member of the leiomodin family. They conducted an observation of this kind of protein for multiple patients in a Lmod2 lab, and the resultant mutant lost its ability to nucleates the actin filaments. This is the reason why mutation can be dangerous to patients, as Wang said.
This work and study could help treat the nemaline myopathy as mutations are addressed.