2016 MDF Fellowship Grant Recipient
Lukasz Sznajder, PhD
Myotonic Dystrophy Type 2 – Mouse Models, Pathomechanism and Therapy
Dr. Sznajder, in his proposal “Myotonic dystrophy type 2: mouse models, pathomechanism and therapy” plans to use recently developed techniques in genetic engineering to generate novel mouse models for DM2. These new mouse models will be thoroughly characterized to determine if they faithfully recapitulate DM2 disease manifestations and to study the molecular events that underlie DM2 disease progression. This DM2 mouse model will be used to develop a therapy for DM2 based on drugs called antisense oligonucleotides, which are currently being tested in clinical trials for DM1.
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