Synthetic polynucleotides encoding a human FKRP protein
AssigneesGenethon, Universite D'Evry Val D'Essonne, Inserm (Institut National De La Sante Et De La Recherche Medicale)
InventorsIsabelle Richard, Evelyne Gicquel-Zouida, William Lostal
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Abstract
The present invention concerns synthetic polynucleotides encoding a human fukutin-related protein (FKRP) wherein the synthetic polynucleotides contain at least a mutation avoiding supplementary transcript(s) generated from frameshift start codon(s). The synthetic polynucleotides are useful, especially for treating a pathology linked to a FKRP deficiency or induced by a defect in α-dystroglycan (α-DG) glycosylation, such as LGMD2I.
Claims12
A synthetic polynucleotide encoding a human fukutin-related protein (FKRP) consisting of or comprising the sequence SEQ ID NO:
- 1, wherein the polynucleotide has at least one antisense start codon mutated, said antisense start codon being located at position 819 of sequence SEQ ID NO:
- 2, wherein the polynucleotide comprises the sequence SEQ ID NO:
- 4.
Dependencies are inferred from the claim text.