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