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COMPOSITIONS AND METHODS FOR CORRECTION OF HERITABLE OCULAR DISEASE
专利权人:
发明人:
申请号:
EP16867296.2
公开号:
EP3377116A1
申请日:
2016.11.18
申请国别(地区):
EP
年份:
2018
代理人:
摘要:
A nucleic acid trans-splicing molecule is provided that can replace an exon in a targeted mammalian ocular gene carrying a defect or mutation causing an ocular disease with an exon having the naturally-occurring sequence without the defect or mutation. A method of treating an ocular disease, e.g., Stargardts Disease, caused by a defect or mutation in a target gene, e.g., ABCA4 comprising: administering to the ocular cells of a subject having an ocular disease a composition comprising a recombinant AAV comprising a nucleic acid trans-splicing molecule as described above.
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