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US 12,104,163 B2Granted
Adeno-associated virus vectors for treatment of Rett syndrome
AssigneeSarepta Therapeutics, Inc.
InventorColin O'Banion
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Abstract
The disclosure provides nucleic acids (comprising AAV expression cassettes), AAV vectors, and compositions for use in methods for treating and/or delaying the onset of diseases associated with mutations in the mecp2 gene, such as Rett Syndrome. Also, provided herein are methods for treating and/or delaying the onset of brain-derived neurotrophic factor (BDNF)-associated diseases.
Claims27
A nucleic acid comprising an adeno-associated virus (AAV) expression cassette, wherein the AAV expression cassette comprises, from 5′ to 3′:
- a 5′ inverted terminal repeat (ITR);
- a minimal human Arc gene promoter (hArcMin) set forth by SEQ ID no. 12;
- a Rett Syndrome-associated gene;
- and
- a 3′ ITR.
2 more elements
Dependencies are inferred from the claim text.