MedicineAmoasii Leonela
Main achievement
Director of Research, Gene Editing, Exonics Therapeutics
About
Leonela Amoasii is an outstanding researcher in the field of molecular biology and gene editing, with major contributions to the development of therapies for Duchenne muscular dystrophy. Born in the Republic of Moldova, she began her academic career at the Institute of Genetics and Molecular and Cellular Biology (IGBMC) at the University of Strasbourg, France, where she defended her PhD, revealing the mechanisms of myotubular centronuclear myopathy and demonstrating expertise in utilizing adeno-associated viruses to manipulate gene expression in muscle. Her work, published in prestigious journals such as Science, has accumulated thousands of citations, including pioneering studies in CRISPR/Cas9 genome editing that restores dystrophin expression in murine and canine models of muscular dystrophy. After her PhD, Leonela Amoasii joined Professor Eric Olson's laboratory as a postdoctoral fellow at UT Southwestern Medical Center in Dallas, Texas, where she optimized in vivo gene editing techniques, achieving promising results for translation into human treatments. She was a founding member of the Exonics Therapeutics team, contributing to building the Boston laboratories and developing gene editing strategies for genetic disorders. Since 2019, she has held the position of Director of Research in Gene Editing at Vertex Pharmaceuticals, following the acquisition of Exonics, and organizes international conferences such as Precision Genome Engineering. With an international career marked by experiences in Moldova, France, and the United States, Leonela speaks five languages – Romanian, Russian, English, French, and Italian – and is recognized for her passion for innovation in regenerative medicine. In her free time, she explores new places, enjoys hiking, cycling, volleyball, and tennis, and dedicates her evenings to music, reading, and photography, reflecting a harmonious balance between science and personal life.