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US 2019/0290781 A1Published application
Methods and Compositions for Treating Dystroglycanopathy Disorders
AssigneesThe Charolotte-Mecklenburg Hospital Authority D/B/A Atrium Health, The University of North Carolina at Chapel Hill
InventorsQi Long Lu, Xiao Xiao
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Abstract
The invention relates to synthetic polynucleotides encoding fukutin related protein (FKRP). The invention further relates to nucleic acid constructs comprising the synthetic polynucleotides and methods of using these synthetic polynucleotides to treat dystroglycanopathy disorders.
Claims20
A polynucleotide comprising a synthetic nucleotide sequence which has at least 90% identity to SEQ ID NO:1 and encodes a human fukutin-related protein (FKRP).
Dependencies are inferred from the claim text.