Back to results
US 2026/0250712 A1Published application
Induction of Immune Tolerance by AAV Vector Comprising the Combination of a Liver Detargeted Capsid and a Tandem Liver-Muscle Specific Promoter
AssigneesGenethon, Universite D'Evry Val D'Essonne, Institut National de la Sante et de la Recherche Medicale
InventorsGiuseppe RONZITTI, Pauline VIDAL
Life of this patent
Estimating the term…
Abstract
The invention relates to an AAV vector comprising the combination of a tandem liver-muscle selective promoter and a liver detargeted AAV capsid protein modified with a muscle-targeting peptide and its use in gene therapy of muscle diseases, in particular genetic neuromuscular diseases.
Claims21
An adeno-associated virus (AAV) vector comprising:
- (i) a transgene of interest operably linked to a tandem promoter comprising a muscle-selective promoter fused to a liver-selective promoter;
- and
- (ii) a peptide-modified AAV capsid protein which comprises the insertion of a muscle-targeting peptide comprising a RGD motif into the variable region VIII of AAV capsid protein sequence, wherein the AAV capsid is detargeted from the liver as the result of the peptide-insertion.
- (iii)
1 more element
3-16. (canceled)
Dependencies are inferred from the claim text.