Anti-CTSA monoclonal antibody, clone FQS21545

Galactosialidosis is a lysosomal storage disease caused by deficiency of protective protein that is encoded by the cathepsin A (CTSA) gene localized on chromosome 20q13.1. Mutations of this gene are the cause of galactosialidosis that result in loss of function of protective protein. Galactosialidosis is an autosomal recessive inherited disease and has been divided into three subtypes based on age of onset and the severity of clinical manifestations. We report an early infantile form of galactosialidosis in a newborn with a novel mutation on the CTSA gene.

Background: The National Institutes of Health funded Clinical and Translational Science Awards (CTSA) have increasingly focused on community-engaged research and funded investigators for community-based participatory research (CBPR). However, because CBPR is a collaborative process focused on community-identified research topics, the Harvard CTSA and its Community Advisory Board (CERAB) funded community partners through a CBPR initiative. Objectives: We describe lessons learned from this seed grants initiative designed to stimulate community academic CBPR partnerships. Methods: The CBPR program of the Harvard CTSA and the CERAB developed this initiative and each round incorporated participant and advisory feedback toward program improvement. Lessons Learned: Although this initiative facilitated relevant and innovative research, challenges included variable community research readiness, insufficient project time, and difficulties identifying investigators for new partnerships. Conclusion: Seed grants can foster innovative CBPR projects. Similar initiatives should consider preliminary assessments of community research readiness as well as strategies for meaningful academic researcher engagement.