Intrathecal delivery of nucleic acid sequences encoding ABCD1 for treatment of adrenomyeloneuropathy
AssigneeThe General Hospital Corporation
InventorsCasey A. Maguire, Florian Eichler
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Abstract
Methods of the invention encompass delivery of nucleic acid sequences encoding ABCD1 for the treatment of X-linked Adrenoleukodystrophy (X-ALD), e.g., for Adrenomyeloneuropathy (AMN).
Claims10
A nucleic acid expression cassette, comprising in order, an inverted terminal repeat (ITR), a cytomegalovirus immediate early (CMV IE) enhancer, a chicken beta-actin promoter, a beta-actin exon, a chimeric intron, a nucleic acid sequence encoding a functional ABCD1 protein, wherein the nucleic acid sequence encoding a functional ABCD1 protein comprises SEQ ID NO:
- 8, a woodchuck hepatitis virus posttranscriptional regulatory element (WPRE), an SV40 poly-A sequence, a bovine growth hormone (BGH) poly-A sequence, and an ITR.
An AAV vector comprising an AAV serotype 9 capsid which has packaged therein a nucleic acid expression cassette comprising in order, an inverted terminal repeat (ITR), a cytomegalovirus immediate early (CMV IE) enhancer, a chicken beta-actin promoter, a beta-actin exon, a chimeric intron, a nucleic acid sequence comprising SEQ ID NO:
- 8 encoding a functional ABCD1 protein, a woodchuck hepatitis virus posttranscriptional regulatory element (WPRE), an SV40 poly-A sequence, a bovine growth hormone (BGH) poly-A sequence, and an ITR.
Dependencies are inferred from the claim text.