COMPOSITIONS AND METHODS FOR TREATMENT OF DISORDERS RELATED TO CEP290

摘要

Compositions are provided that comprise a recombinant vector carrying a nucleic acid sequence encoding a fragment of CEP290 lacking all or part of its N-terminal and C-terminal inhibitory regions, under the control of regulatory sequences which express the product of said gene in a selected cell of a mammalian subject, and a pharmaceutically acceptable carrier. These and other compositions are disclosed with are useful in methods for treating a mammalian subject having a disease associated with a CEP290 mutation, such as Lebers Congenital Amaurosis.

主权项

1. A composition comprising a recombinant vector carrying a nucleic acid sequence encoding a fragment of the CEP290 gene lacking all or part of its N-terminal and C-terminal inhibitory regions, under the control of regulatory sequences which express the product of said nucleic acid sequence in a selected cell of a mammalian subject.