Pitt Pediatrics congratulates Dwi Kemaladewi, PhD, for receiving a R01 grant from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) under the National Institute of Health (NIH). The R01 grant was titled “Therapeutic Genetics and Disease Modeling in LAMA2-deficient Congenital Muscular Dystrophy.”
Kemaladewi’s team in the Division of Genetics and Genomic Medicine plans to use CRISPR activation technology to activate a gene called LAMA1 to compensate for the lack of LAMA2 in mouse and patient derived models, and evaluate the neuromuscular functions, respiratory capability, and immune responses following treatments. Furthermore, they will apply the technology to develop a model to study nerve demyelination, which is largely underexplored in the disease.
Her team will be working alongside John Alcorn, PhD, Quasar Padiath, PhD, and Donna Stolz, PhD. The grant received a 1.0 percentile and was considered more meritorious than 99% of applications peer-reviewed by the committee.