Polynucleotides encoding a human FKRP protein
Grant
US12582723B2
Kind: B2
Mar 24, 2026
Assignee
GENETHON
Inventors
Isabelle Richard, Evelyne Gicquel-Zouida, William Lostal
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.
CPC Classifications
C12N 15/102
C12N 2800/22
C12N 2840/105
A61K 47/549
A61K 48/0091
A61K 48/005
A61K 31/7088
Filing Date
2023-03-02
Application No.
18177446
Claims
9