US2011311506A1PendingUtilityA1

Piggybac transposon variants and methods of use

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Assignee: CRAIG NANCY LYNNPriority: Feb 25, 2009Filed: Feb 25, 2010Published: Dec 22, 2011
Est. expiryFeb 25, 2029(~2.6 yrs left)· nominal 20-yr term from priority
Inventors:Nancy L. Craig
C12N 15/90C12N 2800/00A61K 48/00C12Y 207/07C12N 9/1241A61K 38/45C12N 15/85
48
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Claims

Abstract

The present invention provides hyperactive piggyBac transposons, in particular hyperactive piggyBac transposons from Trichoplusia ni (cabbage looper moth) that transpose at a higher frequency than wildtype. The invention also features integration defective piggyBac transposons. The piggyBac transposons and transposases can be used in gene transfer systems for stably introducing nucleic acids into the DNA of a cell. The gene transfer system can be used in methods, for example, but not limited to, gene therapy, insertional mutagenesis, or gene discovery.

Claims

exact text as granted — not AI-modified
1 . A transposon comprising: one or more hyperactive piggyBac nucleic acid sequences and variants, derivatives and fragments thereof that retain transposon activity. 
     
     
         2 . The transposon of  claim 1 , wherein the hyperactive piggyBac transposon has a higher level of transposon excision compared to a wildtype piggyBac transposon. 
     
     
         3 . The transposon of  claim 1 , comprising 2, 3, 4, 5 or more hyperactive piggyBac nucleic acid sequences and variants, derivatives and fragments thereof that retain transposon activity. 
     
     
         4 . The transposon of  claim 1 , wherein the hyperactive piggyBac nucleic acid sequence is from the family Noctuidae. 
     
     
         5 . The transposon of  claim 1 , wherein the hyperactive piggyBac nucleic acid sequence is from the species  Trichoplusia ni.    
     
     
         6 . The transposon of  claim 1 , comprising a nucleic acid sequence selected from the group consisting of: SEQ ID NO: 34-SEQ ID NO: 63 or SEQ ID NO: 70 SEQ ID NO: 96. 
     
     
         7 . The transposon of  claim 6 , wherein the nucleic acid sequence encodes an amino acid sequence selected from the group consisting of: SEQ ID NO: 3-SEQ ID NO: 32. 
     
     
         8 . A transposon comprising: one or more integration defective piggyBac nucleic acid sequences and variants, derivatives and fragments thereof. 
     
     
         9 . The transposon of  claim 8 , wherein the integration defective piggyBac transposon has a lower rate of integration as compared to a wildtype piggyBac transposon. 
     
     
         10 . The transposon of  claim 8 , wherein the integration defective piggyBac nucleic acid sequence is from the family Noctuidae. 
     
     
         11 . The transposon of  claim 8 , wherein the integration defective piggyBac nucleic acid sequence is from the species  Trichoplusia ni.    
     
     
         12 . The transposon of  claim 8 , comprising a nucleic acid sequence selected from the group consisting of: SEQ ID NO: 67-SEQ ID NO: 69. 
     
     
         13 . The transposon of  claim 12 , wherein the nucleic acid sequence encodes an amino acid sequence selected from the group consisting of: SEQ ID NO: 64-SEQ ID NO: 66. 
     
     
         14 . The transposon of  claim 2 , wherein the wildtype piggyBac transposon comprises a nucleic acid sequence corresponding to SEQ. ID NO: 1. 
     
     
         15 - 24 . (canceled) 
     
     
         25 . A gene transfer system comprising: a transposon according to  claim 1 ; and a piggyBac transposase. 
     
     
         26 - 33 . (canceled) 
     
     
         34 . A cell comprising the transposon of  claim 1 . 
     
     
         35 . A cell comprising the transposon of  claim 8 . 
     
     
         36 . A pharmaceutical composition comprising: a transposon comprising a hyperactive piggyBac nucleic acid sequence and a piggyBac transposase, together with a pharmaceutically acceptable carrier, adjuvant or vehicle. 
     
     
         37 . A method for introducing exogenous DNA into a cell comprising:
 contacting the cell with the gene transfer system of  claim 25 , thereby introducing exogenous DNA into a cell.   
     
     
         38 . The method of  claim 37 , wherein the cell is a stem cell. 
     
     
         39 . A kit comprising: a transposon comprising a hyperactive piggyBac nucleic acid sequence and instructions for introducing DNA into a cell. 
     
     
         40 - 44 . (canceled)

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