METHOD FOR TRANSDUCING Title of Invention CELLS WITH PRIMARY CILIA

Abstract

This invention provides methods for transducing a ciliated cell with a recombinant serotype 2 adeno-associated virus (AAV) vector. Additionally, the invention provides methods of treating diseases associated with a mutated gene by transducing a ciliated cell with a recombinant serotype 2 AAV vector containing a corrective transgene.

1 . A method for transducing a cell comprising primary cilia, comprising the step of: administering to said cell a recombinant serotype 2 adeno-associated virus (AAV) vector; thereby transducing cells comprising primary cilia. 
     
     
         2 . (canceled) 
     
     
         3 . The method of  claim 1 , wherein said cell is a retinal cell. 
     
     
         4 . The method of  claim 1 , wherein said cell is an otocyst cell. 
     
     
         5 . The method of  claim 1 , wherein said cell is a kidney cell. 
     
     
         6 . (canceled) 
     
     
         7 . The method of  claim 1 , wherein said AAV vector comprises a transgene bounded by inverted terminal repeats (ITRs). 
     
     
         8 . (canceled) 
     
     
         9 . A method of treating or inhibiting a kidney disease comprising a defective gene in a subject, comprising the steps of:
 exposing the renal collecting duct of said subject to a recombinant serotype 2 adeno-associated virus (AAV) vector comprising a transgene corrective for said kidney disease; and   delivering said recombinant AAV to a renal tubular epithelial cell;   thereby treating or inhibiting a kidney disease comprising a defective gene in a subject.   
     
     
         10 . The method of  claim 9 , wherein said kidney disease is polycystic kidney disease (PKD). 
     
     
         11 . The method of  claim 9 , wherein said kidney disease is Bardet-Biedl syndrome. 
     
     
         12 . The method of  claim 9 , wherein said kidney disease is Alport syndrome. 
     
     
         13 . The method of  claim 10 , wherein said PKD is autosomal dominant polycystic kidney disease (ADPKD). 
     
     
         14 . (canceled) 
     
     
         15 . The method of  claim 9 , wherein said AAV vector is an AAV serotype 2 packaged in a capsid from AAV serotype 5. 
     
     
         16 . (canceled) 
     
     
         17 . (canceled) 
     
     
         18 . The method of  claim 9 , wherein said delivering comprises retrograde ureteral delivery. 
     
     
         19 . The method of  claim 9 , wherein said treating comprises correcting a pathologic defect. 
     
     
         20 . The method of  claim 9 , wherein said treating comprises reversing a kidney pathology. 
     
     
         21 . The method of  claim 9 , wherein said corrective transgene is pkd1 gene or pkd2 gene. 
     
     
         22 . The method of  claim 9 , wherein said corrective transgene is intraflagellar transport 88 (uft88) gene. 
     
     
         23 .- 27 . (canceled) 
     
     
         28 . A method of preparing a kidney of a subject for transplantation, comprising the steps of:
 exposing the renal collecting duct of said subject; and   delivering said a recombinant serotype 2 adeno-associated virus (AAV) vector comprising a transgene encoding a trophic protein to a renal tubular epithelial cell;   thereby preparing a kidney for transplantation.   
     
     
         29 .- 32 . (canceled) 
     
     
         33 . The method of  claim 28 , wherein said delivering comprises retrograde ureteral delivery. 
     
     
         34 . A method of treating or inhibiting a macular degeneration disease, comprising a mutated fibrillin-like extracellular matrix protein 1 (EFEMP1) gene in a subject comprising the steps of:
 contacting an eye cell with a recombinant serotype 2 adeno-associated virus (AAV) vector comprising a wild-type EFEMP1 transgene;   thereby treating or inhibiting a macular degeneration disease, comprising a mutated fibrillin-like extracellular matrix protein 1 (EFEMP1) gene in a subject.   
     
     
         35 .- 43 . (canceled) 
     
     
         44 . A method for transducing a cochlear cell, comprising the step of:
 administering to said cochlear cell a recombinant serotype 2 adeno-associated virus (AAV) vector;   thereby transducing a cochlear cell.   
     
     
         45 .- 53 . (canceled)