Pitt Pediatrics congratulates assistant professor Dwi Utami Kemaladewi, PhD, of the Division of Genetic and Genomic Medicine on her receipt of a highly competitive New Innovator Award through the National Institutes of Health "High-Risk, High-Reward" research program.
Kemaladewi will lead a project investigating implications of genetic diversity in muscular dystrophy. An individual genetic makeup plays a large role in determining the clinical presentations in many monogenic diseases, including muscular dystrophy. Yet, preclinical studies to evaluate the genetic treatment of monogenic diseases generally lack the in-depth consideration of diverse genetic backgrounds with regards to safety and efficacy. Kemaladewi and colleagues have previously developed a variety of innovative genome editing strategies for the most common form of congenital muscular dystrophy.
The New Innovator Award will support Kemaladewi’s team to incorporate the element of genetic variation in the study of disease mechanisms and therapeutic development. The ability to accurately assess and quantify genetic variant effects on gene therapy approaches is critical for the advancement of the field.
The NIH categorizes this special research program as "outside the box," allowing applicants to pursue innovative proposals that may struggle in standard peer-review processes due to inherent risk.