                         SEQUENCE LISTING

<110>  The Salk Institute for Biological Studies
       The Center for Regenerative Medicine
       Raya, Angel
       Bueren, Juan A.
       Izpisua Belmonte, Juan C.
 
<120>  Generation of Genetically Corrected Disease-Free Induced 
       Pluripotent Stem Cells

<130>  027731-001810PC

<150>  US 61/181287
<151>  2009-05-27

<160>  19    

<170>  PatentIn version 3.3

<210>  1
<211>  360
<212>  PRT
<213>  homo sapiens

<400>  1

Met Ala Gly His Leu Ala Ser Asp Phe Ala Phe Ser Pro Pro Pro Gly 
1               5                   10                  15      


Gly Gly Gly Asp Gly Pro Gly Gly Pro Glu Pro Gly Trp Val Asp Pro 
            20                  25                  30          


Arg Thr Trp Leu Ser Phe Gln Gly Pro Pro Gly Gly Pro Gly Ile Gly 
        35                  40                  45              


Pro Gly Val Gly Pro Gly Ser Glu Val Trp Gly Ile Pro Pro Cys Pro 
    50                  55                  60                  


Pro Pro Tyr Glu Phe Cys Gly Gly Met Ala Tyr Cys Gly Pro Gln Val 
65                  70                  75                  80  


Gly Val Gly Leu Val Pro Gln Gly Gly Leu Glu Thr Ser Gln Pro Glu 
                85                  90                  95      


Gly Glu Ala Gly Val Gly Val Glu Ser Asn Ser Asp Gly Ala Ser Pro 
            100                 105                 110         


Glu Pro Cys Thr Val Thr Pro Gly Ala Val Lys Leu Glu Lys Glu Lys 
        115                 120                 125             


Leu Glu Gln Asn Pro Glu Glu Ser Gln Asp Ile Lys Ala Leu Gln Lys 
    130                 135                 140                 


Glu Leu Glu Gln Phe Ala Lys Leu Leu Lys Gln Lys Arg Ile Thr Leu 
145                 150                 155                 160 


Gly Tyr Thr Gln Ala Asp Val Gly Leu Thr Leu Gly Val Leu Phe Gly 
                165                 170                 175     


Lys Val Phe Ser Gln Thr Thr Ile Cys Arg Phe Glu Ala Leu Gln Leu 
            180                 185                 190         


Ser Phe Lys Asn Met Cys Lys Leu Arg Pro Leu Leu Gln Lys Trp Val 
        195                 200                 205             


Glu Glu Ala Asp Asn Asn Glu Asn Leu Gln Glu Ile Cys Lys Ala Glu 
    210                 215                 220                 


Thr Leu Val Gln Ala Arg Lys Arg Lys Arg Thr Ser Ile Glu Asn Arg 
225                 230                 235                 240 


Val Arg Gly Asn Leu Glu Asn Leu Phe Leu Gln Cys Pro Lys Pro Thr 
                245                 250                 255     


Leu Gln Gln Ile Ser His Ile Ala Gln Gln Leu Gly Leu Glu Lys Asp 
            260                 265                 270         


Val Val Arg Val Trp Phe Cys Asn Arg Arg Gln Lys Gly Lys Arg Ser 
        275                 280                 285             


Ser Ser Asp Tyr Ala Gln Arg Glu Asp Phe Glu Ala Ala Gly Ser Pro 
    290                 295                 300                 


Phe Ser Gly Gly Pro Val Ser Phe Pro Leu Ala Pro Gly Pro His Phe 
305                 310                 315                 320 


Gly Thr Pro Gly Tyr Gly Ser Pro His Phe Thr Ala Leu Tyr Ser Ser 
                325                 330                 335     


Val Pro Phe Pro Glu Gly Glu Ala Phe Pro Pro Val Ser Val Thr Thr 
            340                 345                 350         


Leu Gly Ser Pro Met His Ser Asn 
        355                 360 


<210>  2
<211>  265
<212>  PRT
<213>  homo sapiens

<400>  2

Met His Phe Tyr Arg Leu Phe Leu Gly Ala Thr Arg Arg Phe Leu Asn 
1               5                   10                  15      


Pro Glu Trp Lys Gly Glu Ile Asp Asn Trp Cys Val Tyr Val Leu Thr 
            20                  25                  30          


Ser Leu Leu Pro Phe Lys Ile Gln Ser Gln Asp Ile Lys Ala Leu Gln 
        35                  40                  45              


Lys Glu Leu Glu Gln Phe Ala Lys Leu Leu Lys Gln Lys Arg Ile Thr 
    50                  55                  60                  


Leu Gly Tyr Thr Gln Ala Asp Val Gly Leu Thr Leu Gly Val Leu Phe 
65                  70                  75                  80  


Gly Lys Val Phe Ser Gln Thr Thr Ile Cys Arg Phe Glu Ala Leu Gln 
                85                  90                  95      


Leu Ser Phe Lys Asn Met Cys Lys Leu Arg Pro Leu Leu Gln Lys Trp 
            100                 105                 110         


Val Glu Glu Ala Asp Asn Asn Glu Asn Leu Gln Glu Ile Cys Lys Ala 
        115                 120                 125             


Glu Thr Leu Val Gln Ala Arg Lys Arg Lys Arg Thr Ser Ile Glu Asn 
    130                 135                 140                 


Arg Val Arg Gly Asn Leu Glu Asn Leu Phe Leu Gln Cys Pro Lys Pro 
145                 150                 155                 160 


Thr Leu Gln Gln Ile Ser His Ile Ala Gln Gln Leu Gly Leu Glu Lys 
                165                 170                 175     


Asp Val Val Arg Val Trp Phe Cys Asn Arg Arg Gln Lys Gly Lys Arg 
            180                 185                 190         


Ser Ser Ser Asp Tyr Ala Gln Arg Glu Asp Phe Glu Ala Ala Gly Ser 
        195                 200                 205             


Pro Phe Ser Gly Gly Pro Val Ser Phe Pro Leu Ala Pro Gly Pro His 
    210                 215                 220                 


Phe Gly Thr Pro Gly Tyr Gly Ser Pro His Phe Thr Ala Leu Tyr Ser 
225                 230                 235                 240 


Ser Val Pro Phe Pro Glu Gly Glu Ala Phe Pro Pro Val Ser Val Thr 
                245                 250                 255     


Thr Leu Gly Ser Pro Met His Ser Asn 
            260                 265 


<210>  3
<211>  190
<212>  PRT
<213>  homo sapiens

<400>  3

Met Gly Val Leu Phe Gly Lys Val Phe Ser Gln Thr Thr Ile Cys Arg 
1               5                   10                  15      


Phe Glu Ala Leu Gln Leu Ser Phe Lys Asn Met Cys Lys Leu Arg Pro 
            20                  25                  30          


Leu Leu Gln Lys Trp Val Glu Glu Ala Asp Asn Asn Glu Asn Leu Gln 
        35                  40                  45              


Glu Ile Cys Lys Ala Glu Thr Leu Val Gln Ala Arg Lys Arg Lys Arg 
    50                  55                  60                  


Thr Ser Ile Glu Asn Arg Val Arg Gly Asn Leu Glu Asn Leu Phe Leu 
65                  70                  75                  80  


Gln Cys Pro Lys Pro Thr Leu Gln Gln Ile Ser His Ile Ala Gln Gln 
                85                  90                  95      


Leu Gly Leu Glu Lys Asp Val Val Arg Val Trp Phe Cys Asn Arg Arg 
            100                 105                 110         


Gln Lys Gly Lys Arg Ser Ser Ser Asp Tyr Ala Gln Arg Glu Asp Phe 
        115                 120                 125             


Glu Ala Ala Gly Ser Pro Phe Ser Gly Gly Pro Val Ser Phe Pro Leu 
    130                 135                 140                 


Ala Pro Gly Pro His Phe Gly Thr Pro Gly Tyr Gly Ser Pro His Phe 
145                 150                 155                 160 


Thr Ala Leu Tyr Ser Ser Val Pro Phe Pro Glu Gly Glu Ala Phe Pro 
                165                 170                 175     


Pro Val Ser Val Thr Thr Leu Gly Ser Pro Met His Ser Asn 
            180                 185                 190 


<210>  4
<211>  317
<212>  PRT
<213>  homo sapiens

<400>  4

Met Tyr Asn Met Met Glu Thr Glu Leu Lys Pro Pro Gly Pro Gln Gln 
1               5                   10                  15      


Thr Ser Gly Gly Gly Gly Gly Asn Ser Thr Ala Ala Ala Ala Gly Gly 
            20                  25                  30          


Asn Gln Lys Asn Ser Pro Asp Arg Val Lys Arg Pro Met Asn Ala Phe 
        35                  40                  45              


Met Val Trp Ser Arg Gly Gln Arg Arg Lys Met Ala Gln Glu Asn Pro 
    50                  55                  60                  


Lys Met His Asn Ser Glu Ile Ser Lys Arg Leu Gly Ala Glu Trp Lys 
65                  70                  75                  80  


Leu Leu Ser Glu Thr Glu Lys Arg Pro Phe Ile Asp Glu Ala Lys Arg 
                85                  90                  95      


Leu Arg Ala Leu His Met Lys Glu His Pro Asp Tyr Lys Tyr Arg Pro 
            100                 105                 110         


Arg Arg Lys Thr Lys Thr Leu Met Lys Lys Asp Lys Tyr Thr Leu Pro 
        115                 120                 125             


Gly Gly Leu Leu Ala Pro Gly Gly Asn Ser Met Ala Ser Gly Val Gly 
    130                 135                 140                 


Val Gly Ala Gly Leu Gly Ala Gly Val Asn Gln Arg Met Asp Ser Tyr 
145                 150                 155                 160 


Ala His Met Asn Gly Trp Ser Asn Gly Ser Tyr Ser Met Met Gln Asp 
                165                 170                 175     


Gln Leu Gly Tyr Pro Gln His Pro Gly Leu Asn Ala His Gly Ala Ala 
            180                 185                 190         


Gln Met Gln Pro Met His Arg Tyr Asp Val Ser Ala Leu Gln Tyr Asn 
        195                 200                 205             


Ser Met Thr Ser Ser Gln Thr Tyr Met Asn Gly Ser Pro Thr Tyr Ser 
    210                 215                 220                 


Met Ser Tyr Ser Gln Gln Gly Thr Pro Gly Met Ala Leu Gly Ser Met 
225                 230                 235                 240 


Gly Ser Val Val Lys Ser Glu Ala Ser Ser Ser Pro Pro Val Val Thr 
                245                 250                 255     


Ser Ser Ser His Ser Arg Ala Pro Cys Gln Ala Gly Asp Leu Arg Asp 
            260                 265                 270         


Met Ile Ser Met Tyr Leu Pro Gly Ala Glu Val Pro Glu Pro Ala Ala 
        275                 280                 285             


Pro Ser Arg Leu His Met Ser Gln His Tyr Gln Ser Gly Pro Val Pro 
    290                 295                 300                 


Gly Thr Ala Ile Asn Gly Thr Leu Pro Leu Ser His Met 
305                 310                 315         


<210>  5
<211>  479
<212>  PRT
<213>  homo sapiens

<400>  5

Met Arg Gln Pro Pro Gly Glu Ser Asp Met Ala Val Ser Asp Ala Leu 
1               5                   10                  15      


Leu Pro Ser Phe Ser Thr Phe Ala Ser Gly Pro Ala Gly Arg Glu Lys 
            20                  25                  30          


Thr Leu Arg Gln Ala Gly Ala Pro Asn Asn Arg Trp Arg Glu Glu Leu 
        35                  40                  45              


Ser His Met Lys Arg Leu Pro Pro Val Leu Pro Gly Arg Pro Tyr Asp 
    50                  55                  60                  


Leu Ala Ala Ala Thr Val Ala Thr Asp Leu Glu Ser Gly Gly Ala Gly 
65                  70                  75                  80  


Ala Ala Cys Gly Gly Ser Asn Leu Ala Pro Leu Pro Arg Arg Glu Thr 
                85                  90                  95      


Glu Glu Phe Asn Asp Leu Leu Asp Leu Asp Phe Ile Leu Ser Asn Ser 
            100                 105                 110         


Leu Thr His Pro Pro Glu Ser Val Ala Ala Thr Val Ser Ser Ser Ala 
        115                 120                 125             


Ser Ala Ser Ser Ser Ser Ser Pro Ser Ser Ser Gly Pro Ala Ser Ala 
    130                 135                 140                 


Pro Ser Thr Cys Ser Phe Thr Tyr Pro Ile Arg Ala Gly Asn Asp Pro 
145                 150                 155                 160 


Gly Val Ala Pro Gly Gly Thr Gly Gly Gly Leu Leu Tyr Gly Arg Glu 
                165                 170                 175     


Ser Ala Pro Pro Pro Thr Ala Pro Phe Asn Leu Ala Asp Ile Asn Asp 
            180                 185                 190         


Val Ser Pro Ser Gly Gly Phe Val Ala Glu Leu Leu Arg Pro Glu Leu 
        195                 200                 205             


Asp Pro Val Tyr Ile Pro Pro Gln Gln Pro Gln Pro Pro Gly Gly Gly 
    210                 215                 220                 


Leu Met Gly Lys Phe Val Leu Lys Ala Ser Leu Ser Ala Pro Gly Ser 
225                 230                 235                 240 


Glu Tyr Gly Ser Pro Ser Val Ile Ser Val Ser Lys Gly Ser Pro Asp 
                245                 250                 255     


Gly Ser His Pro Val Val Val Ala Pro Tyr Asn Gly Gly Pro Pro Arg 
            260                 265                 270         


Thr Cys Pro Lys Ile Lys Gln Glu Ala Val Ser Ser Cys Thr His Leu 
        275                 280                 285             


Gly Ala Gly Pro Pro Leu Ser Asn Gly His Arg Pro Ala Ala His Asp 
    290                 295                 300                 


Phe Pro Leu Gly Arg Gln Leu Pro Ser Arg Thr Thr Pro Thr Leu Gly 
305                 310                 315                 320 


Leu Glu Glu Val Leu Ser Ser Arg Asp Cys His Pro Ala Leu Pro Leu 
                325                 330                 335     


Pro Pro Gly Phe His Pro His Pro Gly Pro Asn Tyr Pro Ser Phe Leu 
            340                 345                 350         


Pro Asp Gln Met Gln Pro Gln Val Pro Pro Leu His Tyr Gln Glu Leu 
        355                 360                 365             


Met Pro Pro Gly Ser Cys Met Pro Glu Glu Pro Lys Pro Lys Arg Gly 
    370                 375                 380                 


Arg Arg Ser Trp Pro Arg Lys Arg Thr Ala Thr His Thr Cys Asp Tyr 
385                 390                 395                 400 


Ala Gly Cys Gly Lys Thr Tyr Thr Lys Ser Ser His Leu Lys Ala His 
                405                 410                 415     


Leu Arg Thr His Thr Gly Glu Lys Pro Tyr His Cys Asp Trp Asp Gly 
            420                 425                 430         


Cys Gly Trp Lys Phe Ala Arg Ser Asp Glu Leu Thr Arg His Tyr Arg 
        435                 440                 445             


Lys His Thr Gly His Arg Pro Phe Gln Cys Gln Lys Cys Asp Arg Ala 
    450                 455                 460                 


Phe Ser Arg Ser Asp His Leu Ala Leu His Met Lys Arg His Phe 
465                 470                 475                 


<210>  6
<211>  454
<212>  PRT
<213>  homo sapiens

<400>  6

Met Asp Phe Phe Arg Val Val Glu Asn Gln Gln Pro Pro Ala Thr Met 
1               5                   10                  15      


Pro Leu Asn Val Ser Phe Thr Asn Arg Asn Tyr Asp Leu Asp Tyr Asp 
            20                  25                  30          


Ser Val Gln Pro Tyr Phe Tyr Cys Asp Glu Glu Glu Asn Phe Tyr Gln 
        35                  40                  45              


Gln Gln Gln Gln Ser Glu Leu Gln Pro Pro Ala Pro Ser Glu Asp Ile 
    50                  55                  60                  


Trp Lys Lys Phe Glu Leu Leu Pro Thr Pro Pro Leu Ser Pro Ser Arg 
65                  70                  75                  80  


Arg Ser Gly Leu Cys Ser Pro Ser Tyr Val Ala Val Thr Pro Phe Ser 
                85                  90                  95      


Leu Arg Gly Asp Asn Asp Gly Gly Gly Gly Ser Phe Ser Thr Ala Asp 
            100                 105                 110         


Gln Leu Glu Met Val Thr Glu Leu Leu Gly Gly Asp Met Val Asn Gln 
        115                 120                 125             


Ser Phe Ile Cys Asp Pro Asp Asp Glu Thr Phe Ile Lys Asn Ile Ile 
    130                 135                 140                 


Ile Gln Asp Cys Met Trp Ser Gly Phe Ser Ala Ala Ala Lys Leu Val 
145                 150                 155                 160 


Ser Glu Lys Leu Ala Ser Tyr Gln Ala Ala Arg Lys Asp Ser Gly Ser 
                165                 170                 175     


Pro Asn Pro Ala Arg Gly His Ser Val Cys Ser Thr Ser Ser Leu Tyr 
            180                 185                 190         


Leu Gln Asp Leu Ser Ala Ala Ala Ser Glu Cys Ile Asp Pro Ser Val 
        195                 200                 205             


Val Phe Pro Tyr Pro Leu Asn Asp Ser Ser Ser Pro Lys Ser Cys Ala 
    210                 215                 220                 


Ser Gln Asp Ser Ser Ala Phe Ser Pro Ser Ser Asp Ser Leu Leu Ser 
225                 230                 235                 240 


Ser Thr Glu Ser Ser Pro Gln Gly Ser Pro Glu Pro Leu Val Leu His 
                245                 250                 255     


Glu Glu Thr Pro Pro Thr Thr Ser Ser Asp Ser Glu Glu Glu Gln Glu 
            260                 265                 270         


Asp Glu Glu Glu Ile Asp Val Val Ser Val Glu Lys Arg Gln Ala Pro 
        275                 280                 285             


Gly Lys Arg Ser Glu Ser Gly Ser Pro Ser Ala Gly Gly His Ser Lys 
    290                 295                 300                 


Pro Pro His Ser Pro Leu Val Leu Lys Arg Cys His Val Ser Thr His 
305                 310                 315                 320 


Gln His Asn Tyr Ala Ala Pro Pro Ser Thr Arg Lys Asp Tyr Pro Ala 
                325                 330                 335     


Ala Lys Arg Val Lys Leu Asp Ser Val Arg Val Leu Arg Gln Ile Ser 
            340                 345                 350         


Asn Asn Arg Lys Cys Thr Ser Pro Arg Ser Ser Asp Thr Glu Glu Asn 
        355                 360                 365             


Val Lys Arg Arg Thr His Asn Val Leu Glu Arg Gln Arg Arg Asn Glu 
    370                 375                 380                 


Leu Lys Arg Ser Phe Phe Ala Leu Arg Asp Gln Ile Pro Glu Leu Glu 
385                 390                 395                 400 


Asn Asn Glu Lys Ala Pro Lys Val Val Ile Leu Lys Lys Ala Thr Ala 
                405                 410                 415     


Tyr Ile Leu Ser Val Gln Ala Glu Glu Gln Lys Leu Ile Ser Glu Glu 
            420                 425                 430         


Asp Leu Leu Arg Lys Arg Arg Glu Gln Leu Lys His Lys Leu Glu Gln 
        435                 440                 445             


Leu Arg Asn Ser Cys Ala 
    450                 


<210>  7
<211>  1455
<212>  PRT
<213>  homo sapiens

<400>  7

Met Ser Asp Ser Trp Val Pro Asn Ser Ala Ser Gly Gln Asp Pro Gly 
1               5                   10                  15      


Gly Arg Arg Arg Ala Trp Ala Glu Leu Leu Ala Gly Arg Val Lys Arg 
            20                  25                  30          


Glu Lys Tyr Asn Pro Glu Arg Ala Gln Lys Leu Lys Glu Ser Ala Val 
        35                  40                  45              


Arg Leu Leu Arg Ser His Gln Asp Leu Asn Ala Leu Leu Leu Glu Val 
    50                  55                  60                  


Glu Gly Pro Leu Cys Lys Lys Leu Ser Leu Ser Lys Val Ile Asp Cys 
65                  70                  75                  80  


Asp Ser Ser Glu Ala Tyr Ala Asn His Ser Ser Ser Phe Ile Gly Ser 
                85                  90                  95      


Ala Leu Gln Asp Gln Ala Ser Arg Leu Gly Val Pro Val Gly Ile Leu 
            100                 105                 110         


Ser Ala Gly Met Val Ala Ser Ser Val Gly Gln Ile Cys Thr Ala Pro 
        115                 120                 125             


Ala Glu Thr Ser His Pro Val Leu Leu Thr Val Glu Gln Arg Lys Lys 
    130                 135                 140                 


Leu Ser Ser Leu Leu Glu Phe Ala Gln Tyr Leu Leu Ala His Ser Met 
145                 150                 155                 160 


Phe Ser Arg Leu Ser Phe Cys Gln Glu Leu Trp Lys Ile Gln Ser Ser 
                165                 170                 175     


Leu Leu Leu Glu Ala Val Trp His Leu His Val Gln Gly Ile Val Ser 
            180                 185                 190         


Leu Gln Glu Leu Leu Glu Ser His Pro Asp Met His Ala Val Gly Ser 
        195                 200                 205             


Trp Leu Phe Arg Asn Leu Cys Cys Leu Cys Glu Gln Met Glu Ala Ser 
    210                 215                 220                 


Cys Gln His Ala Asp Val Ala Arg Ala Met Leu Ser Asp Phe Val Gln 
225                 230                 235                 240 


Met Phe Val Leu Arg Gly Phe Gln Lys Asn Ser Asp Leu Arg Arg Thr 
                245                 250                 255     


Val Glu Pro Glu Lys Met Pro Gln Val Thr Val Asp Val Leu Gln Arg 
            260                 265                 270         


Met Leu Ile Phe Ala Leu Asp Ala Leu Ala Ala Gly Val Gln Glu Glu 
        275                 280                 285             


Ser Ser Thr His Lys Ile Val Arg Cys Trp Phe Gly Val Phe Ser Gly 
    290                 295                 300                 


His Thr Leu Gly Ser Val Ile Ser Thr Asp Pro Leu Lys Arg Phe Phe 
305                 310                 315                 320 


Ser His Thr Leu Thr Gln Ile Leu Thr His Ser Pro Val Leu Lys Ala 
                325                 330                 335     


Ser Asp Ala Val Gln Met Gln Arg Glu Trp Ser Phe Ala Arg Thr His 
            340                 345                 350         


Pro Leu Leu Thr Ser Leu Tyr Arg Arg Leu Phe Val Met Leu Ser Ala 
        355                 360                 365             


Glu Glu Leu Val Gly His Leu Gln Glu Val Leu Glu Thr Gln Glu Val 
    370                 375                 380                 


His Trp Gln Arg Val Leu Ser Phe Val Ser Ala Leu Val Val Cys Phe 
385                 390                 395                 400 


Pro Glu Ala Gln Gln Leu Leu Glu Asp Trp Val Ala Arg Leu Met Ala 
                405                 410                 415     


Gln Ala Phe Glu Ser Cys Gln Leu Asp Ser Met Val Thr Ala Phe Leu 
            420                 425                 430         


Val Val Arg Gln Ala Ala Leu Glu Gly Pro Ser Ala Phe Leu Ser Tyr 
        435                 440                 445             


Ala Asp Trp Phe Lys Ala Ser Phe Gly Ser Thr Arg Gly Tyr His Gly 
    450                 455                 460                 


Cys Ser Lys Lys Ala Leu Val Phe Leu Phe Thr Phe Leu Ser Glu Leu 
465                 470                 475                 480 


Val Pro Phe Glu Ser Pro Arg Tyr Leu Gln Val His Ile Leu His Pro 
                485                 490                 495     


Pro Leu Val Pro Gly Lys Tyr Arg Ser Leu Leu Thr Asp Tyr Ile Ser 
            500                 505                 510         


Leu Ala Lys Thr Arg Leu Ala Asp Leu Lys Val Ser Ile Glu Asn Met 
        515                 520                 525             


Gly Leu Tyr Glu Asp Leu Ser Ser Ala Gly Asp Ile Thr Glu Pro His 
    530                 535                 540                 


Ser Gln Ala Leu Gln Asp Val Glu Lys Ala Ile Met Val Phe Glu His 
545                 550                 555                 560 


Thr Gly Asn Ile Pro Val Thr Val Met Glu Ala Ser Ile Phe Arg Arg 
                565                 570                 575     


Pro Tyr Tyr Val Ser His Phe Leu Pro Ala Leu Leu Thr Pro Arg Val 
            580                 585                 590         


Leu Pro Lys Val Pro Asp Ser Arg Val Ala Phe Ile Glu Ser Leu Lys 
        595                 600                 605             


Arg Ala Asp Lys Ile Pro Pro Ser Leu Tyr Ser Thr Tyr Cys Gln Ala 
    610                 615                 620                 


Cys Ser Ala Ala Glu Glu Lys Pro Glu Asp Ala Ala Leu Gly Val Arg 
625                 630                 635                 640 


Ala Glu Pro Asn Ser Ala Glu Glu Pro Leu Gly Gln Leu Thr Ala Ala 
                645                 650                 655     


Leu Gly Glu Leu Arg Ala Ser Met Thr Asp Pro Ser Gln Arg Asp Val 
            660                 665                 670         


Ile Ser Ala Gln Val Ala Val Ile Ser Glu Arg Leu Arg Ala Val Leu 
        675                 680                 685             


Gly His Asn Glu Asp Asp Ser Ser Val Glu Ile Ser Lys Ile Gln Leu 
    690                 695                 700                 


Ser Ile Asn Thr Pro Arg Leu Glu Pro Arg Glu His Met Ala Val Asp 
705                 710                 715                 720 


Leu Leu Leu Thr Ser Phe Cys Gln Asn Leu Met Ala Ala Ser Ser Val 
                725                 730                 735     


Ala Pro Pro Glu Arg Gln Gly Pro Trp Ala Ala Leu Phe Val Arg Thr 
            740                 745                 750         


Met Cys Gly Arg Val Leu Pro Ala Val Leu Thr Arg Leu Cys Gln Leu 
        755                 760                 765             


Leu Arg His Gln Gly Pro Ser Leu Ser Ala Pro His Val Leu Gly Leu 
    770                 775                 780                 


Ala Ala Leu Ala Val His Leu Gly Glu Ser Arg Ser Ala Leu Pro Glu 
785                 790                 795                 800 


Val Asp Val Gly Pro Pro Ala Pro Gly Ala Gly Leu Pro Val Pro Ala 
                805                 810                 815     


Leu Phe Asp Ser Leu Leu Thr Cys Arg Thr Arg Asp Ser Leu Phe Phe 
            820                 825                 830         


Cys Leu Lys Phe Cys Thr Ala Ala Ile Ser Tyr Ser Leu Cys Lys Phe 
        835                 840                 845             


Ser Ser Gln Ser Arg Asp Thr Leu Cys Ser Cys Leu Ser Pro Gly Leu 
    850                 855                 860                 


Ile Lys Lys Phe Gln Phe Leu Met Phe Arg Leu Phe Ser Glu Ala Arg 
865                 870                 875                 880 


Gln Pro Leu Ser Glu Glu Asp Val Ala Ser Leu Ser Trp Arg Pro Leu 
                885                 890                 895     


His Leu Pro Ser Ala Asp Trp Gln Arg Ala Ala Leu Ser Leu Trp Thr 
            900                 905                 910         


His Arg Thr Phe Arg Glu Val Leu Lys Glu Glu Asp Val His Leu Thr 
        915                 920                 925             


Tyr Gln Asp Trp Leu His Leu Glu Leu Glu Ile Gln Pro Glu Ala Asp 
    930                 935                 940                 


Ala Leu Ser Asp Thr Glu Arg Gln Asp Phe His Gln Trp Ala Ile His 
945                 950                 955                 960 


Glu His Phe Leu Pro Glu Ser Ser Ala Ser Gly Gly Cys Asp Gly Asp 
                965                 970                 975     


Leu Gln Ala Ala Cys Thr Ile Leu Val Asn Ala Leu Met Asp Phe His 
            980                 985                 990         


Gln Ser Ser Arg Ser Tyr Asp His  Ser Glu Asn Ser Asp  Leu Val Phe 
        995                 1000                 1005             


Gly Gly  Arg Thr Gly Asn Glu  Asp Ile Ile Ser Arg  Leu Gln Glu 
    1010                 1015                 1020             


Met Val  Ala Asp Leu Glu Leu  Gln Gln Asp Leu Ile  Val Pro Leu 
    1025                 1030                 1035             


Gly His  Thr Pro Ser Gln Glu  His Phe Leu Phe Glu  Ile Phe Arg 
    1040                 1045                 1050             


Arg Arg  Leu Gln Ala Leu Thr  Ser Gly Trp Ser Val  Ala Ala Ser 
    1055                 1060                 1065             


Leu Gln  Arg Gln Arg Glu Leu  Leu Met Tyr Lys Arg  Ile Leu Leu 
    1070                 1075                 1080             


Arg Leu  Pro Ser Ser Val Leu  Cys Gly Ser Ser Phe  Gln Ala Glu 
    1085                 1090                 1095             


Gln Pro  Ile Thr Ala Arg Cys  Glu Gln Phe Phe His  Leu Val Asn 
    1100                 1105                 1110             


Ser Glu  Met Arg Asn Phe Cys  Ser His Gly Gly Ala  Leu Thr Gln 
    1115                 1120                 1125             


Asp Ile  Thr Ala His Phe Phe  Arg Gly Leu Leu Asn  Ala Cys Leu 
    1130                 1135                 1140             


Arg Ser  Arg Asp Pro Ser Leu  Met Val Asp Phe Ile  Leu Ala Lys 
    1145                 1150                 1155             


Cys Gln  Thr Lys Cys Pro Leu  Ile Leu Thr Ser Ala  Leu Val Trp 
    1160                 1165                 1170             


Trp Pro  Ser Leu Glu Pro Val  Leu Leu Cys Arg Trp  Arg Arg His 
    1175                 1180                 1185             


Cys Gln  Ser Pro Leu Pro Arg  Glu Leu Gln Lys Leu  Gln Glu Gly 
    1190                 1195                 1200             


Arg Gln  Phe Ala Ser Asp Phe  Leu Ser Pro Glu Ala  Ala Ser Pro 
    1205                 1210                 1215             


Ala Pro  Asn Pro Asp Trp Leu  Ser Ala Ala Ala Leu  His Phe Ala 
    1220                 1225                 1230             


Ile Gln  Gln Val Arg Glu Glu  Asn Ile Arg Lys Gln  Leu Lys Lys 
    1235                 1240                 1245             


Leu Asp  Cys Glu Arg Glu Glu  Leu Leu Val Phe Leu  Phe Phe Phe 
    1250                 1255                 1260             


Ser Leu  Met Gly Leu Leu Ser  Ser His Leu Thr Ser  Asn Ser Thr 
    1265                 1270                 1275             


Thr Asp  Leu Pro Lys Ala Phe  His Val Cys Ala Ala  Ile Leu Glu 
    1280                 1285                 1290             


Cys Leu  Glu Lys Arg Lys Ile  Ser Trp Leu Ala Leu  Phe Gln Leu 
    1295                 1300                 1305             


Thr Glu  Ser Asp Leu Arg Leu  Gly Arg Leu Leu Leu  Arg Val Ala 
    1310                 1315                 1320             


Pro Asp  Gln His Thr Arg Leu  Leu Pro Phe Ala Phe  Tyr Ser Leu 
    1325                 1330                 1335             


Leu Ser  Tyr Phe His Glu Asp  Ala Ala Ile Arg Glu  Glu Ala Phe 
    1340                 1345                 1350             


Leu His  Val Ala Val Asp Met  Tyr Leu Lys Leu Val  Gln Leu Phe 
    1355                 1360                 1365             


Val Ala  Gly Asp Thr Ser Thr  Val Ser Pro Pro Ala  Gly Arg Ser 
    1370                 1375                 1380             


Leu Glu  Leu Lys Gly Gln Gly  Asn Pro Val Glu Leu  Ile Thr Lys 
    1385                 1390                 1395             


Ala Arg  Leu Phe Leu Leu Gln  Leu Ile Pro Arg Cys  Pro Lys Lys 
    1400                 1405                 1410             


Ser Phe  Ser His Val Ala Glu  Leu Leu Ala Asp Arg  Gly Asp Cys 
    1415                 1420                 1425             


Asp Pro  Glu Val Ser Ala Ala  Leu Gln Ser Arg Gln  Gln Ala Ala 
    1430                 1435                 1440             


Pro Asp  Ala Asp Leu Ser Gln  Glu Pro His Leu Phe  
    1445                 1450                 1455 


<210>  8
<211>  1471
<212>  PRT
<213>  homo sapiens

<400>  8

Met Val Ser Lys Arg Arg Leu Ser Lys Ser Glu Asp Lys Glu Ser Leu 
1               5                   10                  15      


Thr Glu Asp Ala Ser Lys Thr Arg Lys Gln Pro Leu Ser Lys Lys Thr 
            20                  25                  30          


Lys Lys Ser His Ile Ala Asn Glu Val Glu Glu Asn Asp Ser Ile Phe 
        35                  40                  45              


Val Lys Leu Leu Lys Ile Ser Gly Ile Ile Leu Lys Thr Gly Glu Ser 
    50                  55                  60                  


Gln Asn Gln Leu Ala Val Asp Gln Ile Ala Phe Gln Lys Lys Leu Phe 
65                  70                  75                  80  


Gln Thr Leu Arg Arg His Pro Ser Tyr Pro Lys Ile Ile Glu Glu Phe 
                85                  90                  95      


Val Ser Gly Leu Glu Ser Tyr Ile Glu Asp Glu Asp Ser Phe Arg Asn 
            100                 105                 110         


Cys Leu Leu Ser Cys Glu Arg Leu Gln Asp Glu Glu Ala Ser Met Gly 
        115                 120                 125             


Ala Ser Tyr Ser Lys Ser Leu Ile Lys Leu Leu Leu Gly Ile Asp Ile 
    130                 135                 140                 


Leu Gln Pro Ala Ile Ile Lys Thr Leu Phe Glu Lys Leu Pro Glu Tyr 
145                 150                 155                 160 


Phe Phe Glu Asn Lys Asn Ser Asp Glu Ile Asn Ile Pro Arg Leu Ile 
                165                 170                 175     


Val Ser Gln Leu Lys Trp Leu Asp Arg Val Val Asp Gly Lys Asp Leu 
            180                 185                 190         


Thr Thr Lys Ile Met Gln Leu Ile Ser Ile Ala Pro Glu Asn Leu Gln 
        195                 200                 205             


His Asp Ile Ile Thr Ser Leu Pro Glu Ile Leu Gly Asp Ser Gln His 
    210                 215                 220                 


Ala Asp Val Gly Lys Glu Leu Ser Asp Leu Leu Ile Glu Asn Thr Ser 
225                 230                 235                 240 


Leu Thr Val Pro Ile Leu Asp Val Leu Ser Ser Leu Arg Leu Asp Pro 
                245                 250                 255     


Asn Phe Leu Leu Lys Val Arg Gln Leu Val Met Asp Lys Leu Ser Ser 
            260                 265                 270         


Ile Arg Leu Glu Asp Leu Pro Val Ile Ile Lys Phe Ile Leu His Ser 
        275                 280                 285             


Val Thr Ala Met Asp Thr Leu Glu Val Ile Ser Glu Leu Arg Glu Lys 
    290                 295                 300                 


Leu Asp Leu Gln His Cys Val Leu Pro Ser Arg Leu Gln Ala Ser Gln 
305                 310                 315                 320 


Val Lys Leu Lys Ser Lys Gly Arg Ala Ser Ser Ser Gly Asn Gln Glu 
                325                 330                 335     


Ser Ser Gly Gln Ser Cys Ile Ile Leu Leu Phe Asp Val Ile Lys Ser 
            340                 345                 350         


Ala Ile Arg Tyr Glu Lys Thr Ile Ser Glu Ala Trp Ile Lys Ala Ile 
        355                 360                 365             


Glu Asn Thr Ala Ser Val Ser Glu His Lys Val Phe Asp Leu Val Met 
    370                 375                 380                 


Leu Phe Ile Ile Tyr Ser Thr Asn Thr Gln Thr Lys Lys Tyr Ile Asp 
385                 390                 395                 400 


Arg Val Leu Arg Asn Lys Ile Arg Ser Gly Cys Ile Gln Glu Gln Leu 
                405                 410                 415     


Leu Gln Ser Thr Phe Ser Val His Tyr Leu Val Leu Lys Asp Met Cys 
            420                 425                 430         


Ser Ser Ile Leu Ser Leu Ala Gln Ser Leu Leu His Ser Leu Asp Gln 
        435                 440                 445             


Ser Ile Ile Ser Phe Gly Ser Leu Leu Tyr Lys Tyr Ala Phe Lys Phe 
    450                 455                 460                 


Phe Asp Thr Tyr Cys Gln Gln Glu Val Val Gly Ala Leu Val Thr His 
465                 470                 475                 480 


Ile Cys Ser Gly Asn Glu Ala Glu Val Asp Thr Ala Leu Asp Val Leu 
                485                 490                 495     


Leu Glu Leu Val Val Leu Asn Pro Ser Ala Met Met Met Asn Ala Val 
            500                 505                 510         


Phe Val Lys Gly Ile Leu Asp Tyr Leu Asp Asn Ile Ser Pro Gln Gln 
        515                 520                 525             


Ile Arg Lys Leu Phe Tyr Val Leu Ser Thr Leu Ala Phe Ser Lys Gln 
    530                 535                 540                 


Asn Glu Ala Ser Ser His Ile Gln Asp Asp Met His Leu Val Ile Arg 
545                 550                 555                 560 


Lys Gln Leu Ser Ser Thr Val Phe Lys Tyr Lys Leu Ile Gly Ile Ile 
                565                 570                 575     


Gly Ala Val Thr Met Ala Gly Ile Met Ala Ala Asp Arg Ser Glu Ser 
            580                 585                 590         


Pro Ser Leu Thr Gln Glu Arg Ala Asn Leu Ser Asp Glu Gln Cys Thr 
        595                 600                 605             


Gln Val Thr Ser Leu Leu Gln Leu Val His Ser Cys Ser Glu Gln Ser 
    610                 615                 620                 


Pro Gln Ala Ser Ala Leu Tyr Tyr Asp Glu Phe Ala Asn Leu Ile Gln 
625                 630                 635                 640 


His Glu Lys Leu Asp Pro Lys Ala Leu Glu Trp Val Gly His Thr Ile 
                645                 650                 655     


Cys Asn Asp Phe Gln Asp Ala Phe Val Val Asp Ser Cys Val Val Pro 
            660                 665                 670         


Glu Gly Asp Phe Pro Phe Pro Val Lys Ala Leu Tyr Gly Leu Glu Glu 
        675                 680                 685             


Tyr Asp Thr Gln Asp Gly Ile Ala Ile Asn Leu Leu Pro Leu Leu Phe 
    690                 695                 700                 


Ser Gln Asp Phe Ala Lys Asp Gly Gly Pro Val Thr Ser Gln Glu Ser 
705                 710                 715                 720 


Gly Gln Lys Leu Val Ser Pro Leu Cys Leu Ala Pro Tyr Phe Arg Leu 
                725                 730                 735     


Leu Arg Leu Cys Val Glu Arg Gln His Asn Gly Asn Leu Glu Glu Ile 
            740                 745                 750         


Asp Gly Leu Leu Asp Cys Pro Ile Phe Leu Thr Asp Leu Glu Pro Gly 
        755                 760                 765             


Glu Lys Leu Glu Ser Met Ser Ala Lys Glu Arg Ser Phe Met Cys Ser 
    770                 775                 780                 


Leu Ile Phe Leu Thr Leu Asn Trp Phe Arg Glu Ile Val Asn Ala Phe 
785                 790                 795                 800 


Cys Gln Glu Thr Ser Pro Glu Met Lys Gly Lys Val Leu Thr Arg Leu 
                805                 810                 815     


Lys His Ile Val Glu Leu Gln Ile Ile Leu Glu Lys Tyr Leu Ala Val 
            820                 825                 830         


Thr Pro Asp Tyr Val Pro Pro Leu Gly Asn Phe Asp Val Glu Thr Leu 
        835                 840                 845             


Asp Ile Thr Pro His Thr Val Thr Ala Ile Ser Ala Lys Ile Arg Lys 
    850                 855                 860                 


Lys Gly Lys Ile Glu Arg Lys Gln Lys Thr Asp Gly Ser Lys Thr Ser 
865                 870                 875                 880 


Ser Ser Asp Thr Leu Ser Glu Glu Lys Asn Ser Glu Cys Asp Pro Thr 
                885                 890                 895     


Pro Ser His Arg Gly Gln Leu Asn Lys Glu Phe Thr Gly Lys Glu Glu 
            900                 905                 910         


Lys Thr Ser Leu Leu Leu His Asn Ser His Ala Phe Phe Arg Glu Leu 
        915                 920                 925             


Asp Ile Glu Val Phe Ser Ile Leu His Cys Gly Leu Val Thr Lys Phe 
    930                 935                 940                 


Ile Leu Asp Thr Glu Met His Thr Glu Ala Thr Glu Val Val Gln Leu 
945                 950                 955                 960 


Gly Pro Pro Glu Leu Leu Phe Leu Leu Glu Asp Leu Ser Gln Lys Leu 
                965                 970                 975     


Glu Ser Met Leu Thr Pro Pro Ile Ala Arg Arg Val Pro Phe Leu Lys 
            980                 985                 990         


Asn Lys Gly Ser Arg Asn Ile Gly  Phe Ser His Leu Gln  Gln Arg Ser 
        995                 1000                 1005             


Ala Gln  Glu Ile Val His Cys  Val Phe Gln Leu Leu  Thr Pro Met 
    1010                 1015                 1020             


Cys Asn  His Leu Glu Asn Ile  His Asn Tyr Phe Gln  Cys Leu Ala 
    1025                 1030                 1035             


Ala Glu  Asn His Gly Val Val  Asp Gly Pro Gly Val  Lys Val Gln 
    1040                 1045                 1050             


Glu Tyr  His Ile Met Ser Ser  Cys Tyr Gln Arg Leu  Leu Gln Ile 
    1055                 1060                 1065             


Phe His  Gly Leu Phe Ala Trp  Ser Gly Phe Ser Gln  Pro Glu Asn 
    1070                 1075                 1080             


Gln Asn  Leu Leu Tyr Ser Ala  Leu His Val Leu Ser  Ser Arg Leu 
    1085                 1090                 1095             


Lys Gln  Gly Glu His Ser Gln  Pro Leu Glu Glu Leu  Leu Ser Gln 
    1100                 1105                 1110             


Ser Val  His Tyr Leu Gln Asn  Phe His Gln Ser Ile  Pro Ser Phe 
    1115                 1120                 1125             


Gln Cys  Ala Leu Tyr Leu Ile  Arg Leu Leu Met Val  Ile Leu Glu 
    1130                 1135                 1140             


Lys Ser  Thr Ala Ser Ala Gln  Asn Lys Glu Lys Ile  Ala Ser Leu 
    1145                 1150                 1155             


Ala Arg  Gln Phe Leu Cys Arg  Val Trp Pro Ser Gly  Asp Lys Glu 
    1160                 1165                 1170             


Lys Ser  Asn Ile Ser Asn Asp  Gln Leu His Ala Leu  Leu Cys Ile 
    1175                 1180                 1185             


Tyr Leu  Glu His Thr Glu Ser  Ile Leu Lys Ala Ile  Glu Glu Ile 
    1190                 1195                 1200             


Ala Gly  Val Gly Val Pro Glu  Leu Ile Asn Ser Pro  Lys Asp Ala 
    1205                 1210                 1215             


Ser Ser  Ser Thr Phe Pro Thr  Leu Thr Arg His Thr  Phe Val Val 
    1220                 1225                 1230             


Phe Phe  Arg Val Met Met Ala  Glu Leu Glu Lys Thr  Val Lys Lys 
    1235                 1240                 1245             


Ile Glu  Pro Gly Thr Ala Ala  Asp Ser Gln Gln Ile  His Glu Glu 
    1250                 1255                 1260             


Lys Leu  Leu Tyr Trp Asn Met  Ala Val Arg Asp Phe  Ser Ile Leu 
    1265                 1270                 1275             


Ile Asn  Leu Ile Lys Val Phe  Asp Ser His Pro Val  Leu His Val 
    1280                 1285                 1290             


Cys Leu  Lys Tyr Gly Arg Leu  Phe Val Glu Ala Phe  Leu Lys Gln 
    1295                 1300                 1305             


Cys Met  Pro Leu Leu Asp Phe  Ser Phe Arg Lys His  Arg Glu Asp 
    1310                 1315                 1320             


Val Leu  Ser Leu Leu Glu Thr  Phe Gln Leu Asp Thr  Arg Leu Leu 
    1325                 1330                 1335             


His His  Leu Cys Gly His Ser  Lys Ile His Gln Asp  Thr Arg Leu 
    1340                 1345                 1350             


Thr Gln  His Val Pro Leu Leu  Lys Lys Thr Leu Glu  Leu Leu Val 
    1355                 1360                 1365             


Cys Arg  Val Lys Ala Met Leu  Thr Leu Asn Asn Cys  Arg Glu Ala 
    1370                 1375                 1380             


Phe Trp  Leu Gly Asn Leu Lys  Asn Arg Asp Leu Gln  Gly Glu Glu 
    1385                 1390                 1395             


Ile Lys  Ser Gln Asn Ser Gln  Glu Ser Thr Ala Asp  Glu Ser Glu 
    1400                 1405                 1410             


Asp Asp  Met Ser Ser Gln Ala  Ser Lys Ser Lys Ala  Thr Glu Val 
    1415                 1420                 1425             


Ser Leu  Gln Asn Pro Pro Glu  Ser Gly Thr Asp Gly  Cys Ile Leu 
    1430                 1435                 1440             


Leu Ile  Val Leu Ser Trp Trp  Ser Arg Thr Leu Pro  Thr Tyr Val 
    1445                 1450                 1455             


Tyr Cys  Gln Met Leu Leu Cys  Pro Phe Pro Phe Pro  Pro 
    1460                 1465                 1470     


<210>  9
<211>  19
<212>  DNA
<213>  Artificial

<220>
<223>  Synthetic DNA primer for FANCA transgene.

<400>  9
gctcaagggt cagggcaac                                                  19


<210>  10
<211>  21
<212>  DNA
<213>  Artificial

<220>
<223>  Synthetic DNA primer for FNACA transgene.

<400>  10
tgtgagaagc tctttttcgg g                                               21


<210>  11
<211>  31
<212>  DNA
<213>  Artificial

<220>
<223>  Synthetic DNA probe to quantify proviral copy number.


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  conjugated to FAM

<220>
<221>  misc_feature
<222>  (31)..(31)
<223>  conjugated to BHQ1

<400>  11
cgtctttttc tgctgcagtt aatacctcgg t                                    31


<210>  12
<211>  19
<212>  DNA
<213>  Artificial

<220>
<223>  Synthetic DNA primer to quantify number of cells.

<400>  12
attggcaatg agcggttcc                                                  19


<210>  13
<211>  21
<212>  DNA
<213>  Artificial

<220>
<223>  Synthetic DNA primer.

<400>  13
acagtctcca ctcacccagg a                                               21


<210>  14
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Synthetic DNA probe.


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  Conjugated with Texas Red.

<220>
<221>  misc_feature
<222>  (25)..(25)
<223>  Conjugated with BHQ1.

<400>  14
ccctgaggca ctcttccagc cttcc                                           25


<210>  15
<211>  19
<212>  DNA
<213>  Artificial

<220>
<223>  Synthetic DNA primer.

<400>  15
tcttctgacg ggacctgcc                                                  19


<210>  16
<211>  29
<212>  DNA
<213>  Artificial

<220>
<223>  Synthetic DNA primer

<400>  16
aagagctcca tgttatgctt gtaataaat                                       29


<210>  17
<211>  24
<212>  DNA
<213>  Artificial

<220>
<223>  Synthetic DNA probe.


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  Conjugated with FAM.

<220>
<221>  misc_feature
<222>  (24)..(24)
<223>  Conjugated with BHQ1.

<400>  17
cacaccagcc cagctcccgt gtaa                                            24


<210>  18
<211>  18
<212>  DNA
<213>  Artificial

<220>
<223>  Synthetic DNA primer.

<400>  18
cacaggactc catgccca                                                   18


<210>  19
<211>  21
<212>  DNA
<213>  Artificial

<220>
<223>  Synthetic Luciferase siRNA having deoxythymidine at positions
       20-21.

<220>
<223>  Description of combined DNA/RNA molecule: synthetic Luciferase  
       siRNA having deoxythymidine at positions 20-21.

<220>
<221>  misc_feature
<222>  (1)..(19)
<223>  Ribonucleotides


<400>  19
cguacgcgga auacuucgat t                                               21


