Nucleic Acid-Based Therapy of Muscular Dystrophies
AssigneeModernaTX, Inc.
InventorPaolo Martini
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Abstract
The invention related to polynucleotides comprising an open reading frame of linked nucleosides encoding therapeutic proteins or variant therapeutic proteins, isoforms thereof, functional fragments thereof, and fusion proteins comprising therapeutic proteins. In some embodiments, the open reading frame is sequence-optimized. The invention also relates to methods of treating muscular dystrophies.
Claims16
-89. (canceled)
A method of treating Duchenne muscular dystrophy in a human subject in need thereof, the method comprising administering to the human subject a therapeutically effective amount of a lipid nanoparticle comprising a messenger RNA (mRNA) encoding a JAG1 polypeptide.
A method of treating Duchenne muscular dystrophy in a human subject in need thereof, the method comprising administering to the human subject a therapeutically effective amount of a lipid nanoparticle comprising a messenger RNA (mRNA) encoding a dystrophin polypeptide.
Dependencies are inferred from the claim text.