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US 2024/0252686 A1Published application
Aav for the gene therapy of wet-amd
AssigneeSkyline Therapeutics Limited
InventorsJinzhao HOU, Yanqun SHU, Qiongqiong REN, Heping LI
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Abstract
The present invention provides a polynucleotide and vector, in particular AAV vector, encoding a nanobody against VEGF. The present invention also provides a method of treating a disease associated with VEGF, such as the overexpression of VEGF, e.g., Wet-AMD and DME comprising administering the vector by, e.g., intravitreal injection to provide stable expression of the nanobody in eyes.
Claims21
A polynucleotide construct comprising an expression cassette comprising a coding sequence comprising a first nucleotide sequence selected from SEQ ID NOs:
- 4, 5 and 7, and a second nucleotide sequence linked to the first nucleotide sequence selected from SEQ ID NOs:
- 15, 16 and 18 via a linker operably linked to a promoter.
Dependencies are inferred from the claim text.