The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Mar. 07, 2023
Filed:
Jul. 06, 2018
Genethon, Evry, FR;
Universite D'evry-val-d'essonne, Evry, FR;
Inserm (Institut National DE LA Sante ET DE LA Recherche Medicale), Paris, FR;
Isabelle Richard, Corbeil-Essonnes, FR;
Evelyne Gicquel-Zouida, Vert-le-Petit, FR;
William Lostal, Savigny sur Orge, FR;
GENETHON, Evry, FR;
UNIVERSITE D'EVRY-VAL-D'ESSONNE, Evry, FR;
INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE), Paris, FR;
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.