RNA-Based therapeutics for OTC deficiency
Balestra, Dario
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Anno: 2018 |
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Elucidation of aberrant SBDS splicing mechanisms to design RNA-therapeutics for Shwachman-Diamond Syndrome
Balestra, Dario
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Anno: 2018 |
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F8 splicing-swithcing molecules for tailored hemophilia A therapies
Balestra, Dario
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Anno: 2017 |
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