Means and methods for the specific modulation of target genes in the CNS and the eye and methods for their identification

摘要

Provided are methods for the treatment of disorders of the central nervous system (CNS) and the eye. In particular, use of compositions comprising a compound capable of modulating a target gene or gene product is described for the preparation of a pharmaceutical composition for the treatment of disorders of the CNS and/or the eye, wherein the composition is designed to be administered outside the blood-CNS and the blood-retina barriers. Furthermore, methods are provided for identifying and obtaining nucleic acid molecules encoding polypeptides involved in CNS disorders or of the eye, methods for diagnosing said disorders as well as transgenic animal deficient in the expression of target genes identified in accordance with the described method. In addition, methods of identifying and isolating drugs that are particularly useful for the treatment of disorders related to the CNS and/or the eye are disclosed.

主权项

1. A method for the treatment of wet age-related macular degeneration or diabetic retinopathy comprising administering to a subject who has been diagnosed with wet age-related macular degeneration or diabetic retinopathy a composition comprising a RNA compound comprising a double stranded region of RNA in a therapeutically effective amount to reduce levels of a target mRNA,
wherein said composition is administered outside the blood-retina barrier and the composition crosses the blood-retina barrier and reduces levels of the target mRNA inside the blood-retina barrier and treats the wet age-related macular degeneration or diabetic retinopathy of the subject who has been diagnosed with wet age-related macular degeneration or diabetic retinopathy, wherein said double stranded region is 21-23 nucleotides in length.