Retinoid replacements and opsin agonists and methods for the use thereof
Compositions of and methods for using synthetic retinoids as retinoid replacements and opsin agonists are provided.
1. A method of treating Leber Congenital Amaurosis (LCA) in an LRAT-defective human subject having a mutation in the gene encoding the LRAT protein, comprising:
administering a therapeutically effective amount of a 9-cis-retinal to the LRAT-defective human subject, wherein the LRAT-defective human subject has a deficiency of endogenous 11-cis-retinal in the eye and the 9-cis-retinal forms a functional opsin/chromophore complex in the eye.
2. The method of claim 1 , wherein the 9-cis-retinal is administered locally to the eye of the human subject.
3. The method of claim 2 , wherein the local administration is by eye drops, intraocular injection or periocular injection.
4. The method of claim 1 wherein the 9-cis-retinal is administered systemically by oral dosage form or injection.
5. A method of treating Leber Congenital Amaurosis (LCA) in an LRAT-defective human subject having a mutation in the gene encoding the LRAT protein, comprising:
administering a therapeutically effective amount of 9-cis-retinal by local administration to an eye of the LRAT-defective human subject, wherein the LRAT-defective human subject has a deficiency of endogenous 11-cis-retinal in the eye and the 9-cis-retinal forms a functional opsin/chromophore complex in the eye.
6. The method of claim 5 , wherein the local administration is by eye drops, intraocular injection or periocular injection.