Method of treating ocular diseases by gene therapy
OwnerAlberto Auricchioinventor
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Abstract
A method for the treatment of diseases associated with mutations in ABCA4 gene by administering, to a subject in need thereof, an adeno-associated viral vector encoding an ABCR protein; genetic constructs and adeno-associated viral vectors for use in this method.
Claims13
A method for correcting retinal abnormalities and/or retinal function in a subject affected by a disease associated with mutations in ABCA4 gene, said method comprising the following steps:
- 1) providing a recombinant adeno-associated viral (AAV) vector with AAV5 capsid, said vector carrying an expression cassette which contains a nucleic acid molecule encoding a functional ABCR protein, wherein said nucleic acid molecule is operably linked to regulatory control elements that direct the transcription and translation thereof;
- 2) transducing photoreceptor cells with said recombinant AAV vector, whereby the expression of the ABCR protein is induced in said cells.
A recombinant adeno-associated viral (AAV) vector with AAV5 capsid carrying an expression cassette in which a coding sequence of ABCA4 is functionally linked to a promoter sequence able to regulate its expression in mammalian retinal cells.
Dependencies are inferred from the claim text.