Intrathecal delivery of recombinant adeno-associated virus encoding methyl-cpg binding protein 2
NZ794471BActive Publication Date: 2026-09-01NATIONWIDE CHILDRENS HOSPITAL +1
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Patent Information
- Application Number
- NZ794471
- Authority / Receiving Office
- NZ · NZ
- Patent Type
- Patents
- Current Assignee / Owner
- Priority Date
- 2016-11-17
- Filing Date
- 2017-11-17
- Publication Date
- 2026-09-01
- Estimated Expiration
- 2037-11-17
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Abstract
Methods and materials for intrathecal delivery of recombinant Adeno-associated virus 9 (rAAV9), the rAAV9 including a genome, the genome including a first AAV2 inverted terminal report (ITR); an SV40 intron; a promoter of SEQ ID NO: 2; a polynucleotide sequence encoding Methyl-CpG binding protein 2 (MECP2) comprising SEQ ID NO: 3, where the promoter is operably linked to the polynucleotide sequence encoding the MECP2; a synthetic polyadenylation signal; and a second AAV2 ITR are provided. Use of the methods and materials is contemplated, for example, for the treatment of Rett syndrome.
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