Aav vector for treatment of friedreich's ataxia
AssigneeUniversity of Florida Research Foundation, Inc.
InventorsManuela Corti, Barry John Byrne
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Abstract
Provided herein are nucleic acids, recombinant adeno-associated viral particles, compositions and methods related to treating Friedreich's ataxia. In some examples, the nucleic acids, recombinant adeno-associated viral particles, compositions and methods involve us of a FXN coding sequence, a truncated FXN 3′ UTR, and a prompter.
Claims23
A nucleic acid comprising an expression construct comprising a human frataxin (FXN) coding sequence and a truncated human FXN 3′ untranslated region (UTR) operably linked to a promoter, wherein the expression construct is flanked on each side by an inverted terminal repeat sequence.
Dependencies are inferred from the claim text.