Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
AssigneesVoyager Therapeutics, Inc., Inserm (Institut National De La Sante Et De La Recherche Medicale), Centre National de la Recherche Scientifique (CNRS), Universite De Strasbourg
InventorsDinah Wen-Yee Sah, Martin Goulet, Holger Patzke, Yanqun Shu, Jinzhao Hou, Hélène Puccio
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Abstract
Described herein are compositions and methods for treating Friedreich's Ataxia (FA) using adeno-associated virus (AAV) to deliver therapeutics agents.
Claims23
An adeno-associated virus (AAV) particle comprising a neurotropic capsid and a viral genome, wherein said viral genome comprises a polynucleotide sequence encoding Frataxin and one or more microRNA binding sites.
A method for treating, ameliorating, and/or preventing a neurological disease in a subject stemming from a loss or partial loss of frataxin protein in the subject, wherein the method comprises:
- administering to the subject a therapeutically effective amount of a composition comprising an AAV particle of any of claims 1-13. 15, The method of claim 14, wherein the AAV particle is administered by intravenous (IV) administration.
Dependencies are inferred from the claim text.