Methods of gene therapy using nucleic acid sequences for atp-binding cassette transporter

Abstract

The present invention provides nucleic acid and amino acid sequences of an ATP binding cassette transporter and mutated sequences thereof associated with macular degeneration. Methods of detecting agents that modify ATP-binding cassette transporter comprising combining purified ATP binding cassette transporter and at least one agent suspected of modifying the ATP binding cassette transporter an observing a change in at least one characteristic associated with ATP binding cassette transporter. Methods of detecting macular degeneration is also embodied by the present invention.

1 .- 6 . (canceled) 
     
     
         7 . A purified nucleic acid molecule comprising a nucleotide sequence encoding an ABCR amino acid sequence of SEQ ID NO:11. 
     
     
         8 . The nucleic acid molecule of  claim 7 , wherein the nucleotide sequence encodes wild-type ABCR. 
     
     
         9 . A vector comprising a nucleotide sequence encoding an ABCR amino acid sequence according to SEQ ID NO: 11. 
     
     
         10 . The vector of  claim 9 , wherein the vector is a viral vector. 
     
     
         11 . The vector of  claim 9 , wherein the nucleotide sequence encodes wild-type ABCR. 
     
     
         12 . An isolated cell comprising a nucleotide sequence encoding an ABCR amino acid sequence of SEQ ID NO:11. 
     
     
         13 . The cell of  claim 12 , wherein the nucleotide sequence encodes wild-type ABCR.