AAV vector for treatment of Friedreich's ataxia
AssigneeUniversity of Florida Research Foundation, Inc.
InventorsManuela Corti, Barry John Byrne
Life of this patent
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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.
Claims22
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,
- wherein the FXN 3′ UTR is truncated relative to the wild-type FXN 3′ UTR sequence of SEQ ID NO:
- 9,
- wherein the FXN coding sequence is codon-optimized for expression in human cells, and
- wherein the FXN coding sequence is at least 90% identical to a sequence within SEQ ID NO:
- 1 that encodes a 210 amino acid human FXN protein.
A nucleic acid comprising an expression construct comprising a codon-optimized 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,
- wherein the FXN coding sequence is codon-optimized for expression in human cells, and
- wherein the wild-type FXN 3′ UTR comprises a nucleic acid sequence of SEQ ID NO:
- 3 and is no more than 2000 nucleotides in length.
A nucleic acid comprising an expression construct comprising a codon-optimized human frataxin (FXN) coding sequence, wherein the expression construct is flanked on each side by an inverted terminal repeat sequence, wherein the FXN coding sequence is codon-optimized for expression in human cells, and wherein the FXN coding sequence is at least 90% identical to a sequence within SEQ ID NO:
- 1 that encodes a 210 amino acid human FXN protein.
Dependencies are inferred from the claim text.