We are happy to present the winners of the American Neuromuscular Foundation’s (ANF) 2024 Investigator Grant Award. Liubov Gushchina, PhD, received the prize for his research effort, “Advancing Care With Exon 17 Skipping Therapy for Duchenne Muscular Dystrophy,” and Brett Morrison, MD, PhD, for her study, “Role of Macrophage MCT1 in Diabetic Peripheral Neuropathy.”
Exon 17 Skipping Study
In order to restore the open-reading frame that will lead to the restoration of dystrophin protein, Dr. Gushchina’s initiative intends to create and evaluate an adeno-associated virus (AAV)-mediated treatment that excludes exon 17 from the Duchenne Muscular Dystrophy (DMD) transcript.
According to Dr. Gushchina, whose research attempts to develop the second generation of AAV vectors with enhanced efficiency and safety profiles in human cells in vitro and in mouse skeletal muscles in vivo, “this very promising approach can be applied to Duchenne Muscular Dystrophy patients carrying skip-amendable mutations (duplications and deletions) and flank exon 17.” [Read more about exon 17 skipping study]
