                         SEQUENCE LISTING

<110>  THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK
 
<120>  DUAL EXPRESSION VECTOR FOR GENE AUGMENTATION FOR CRUMBS COMPLEX 
       HOMOLOGUE 1 (CRB1) MUTATIONS

<130>  COLUM-40032.601

<150>  US 63/123,766
<151>  2020-12-10

<160>  9     

<170>  PatentIn version 3.5

<210>  1
<211>  20
<212>  DNA
<213>  Artificial sequence

<220>
<223>  Synthetic

<400>  1
taacccctgc cagtccaatg                                                   20


<210>  2
<211>  20
<212>  DNA
<213>  Artificial sequence

<220>
<223>  Synthetic

<400>  2
ccacacattc cccattggac                                                   20


<210>  3
<211>  20
<212>  DNA
<213>  Artificial sequence

<220>
<223>  Synthetic

<400>  3
tttggccagg atgactccac                                                   20


<210>  4
<211>  20
<212>  DNA
<213>  Artificial sequence

<220>
<223>  Synthetic

<400>  4
cataaccagt ggagtcatcc                                                   20


<210>  5
<211>  80
<212>  DNA
<213>  Homo sapiens

<400>  5
gacctcaatg aatgcaatag taacccctgc cagtccaatg gggaatgtgt ggagctgtcc       60

tcagagaaac aatatggacg                                                   80


<210>  6
<211>  80
<212>  DNA
<213>  Homo sapiens

<400>  6
aagagtatgt ggcaggcaga tttggccagg atgactccac tggttatgtc atctttactc       60

ttgatgagag ctatggagac                                                   80


<210>  7
<211>  180
<212>  DNA
<213>  Artificial sequence

<220>
<223>  Synthetic

<400>  7
tacacaggtg cccagtgtga gatcgacctc aatgaatgca atagtaaccc ctgccagtcc       60

acactggtta tgtcatcttt actcttgatg agagctatgg agacaccatc agcctctcca      120

tgtttgtccg aacgcttcaa ccatcaggct tacttctagc tttggaaaac agcacttatc      180


<210>  8
<211>  1406
<212>  PRT
<213>  Homo sapiens

<400>  8

Met Ala Leu Lys Asn Ile Asn Tyr Leu Leu Ile Phe Tyr Leu Ser Phe 
1               5                   10                  15      


Ser Leu Leu Ile Tyr Ile Lys Asn Ser Phe Cys Asn Lys Asn Asn Thr 
            20                  25                  30          


Arg Cys Leu Ser Asn Ser Cys Gln Asn Asn Ser Thr Cys Lys Asp Phe 
        35                  40                  45              


Ser Lys Asp Asn Asp Cys Ser Cys Ser Asp Thr Ala Asn Asn Leu Asp 
    50                  55                  60                  


Lys Asp Cys Asp Asn Met Lys Asp Pro Cys Phe Ser Asn Pro Cys Gln 
65                  70                  75                  80  


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


Lys Cys Pro Pro Gly Tyr Ser Gly Thr Ile Cys Glu Thr Thr Ile Gly 
            100                 105                 110         


Ser Cys Gly Lys Asn Ser Cys Gln His Gly Gly Ile Cys His Gln Asp 
        115                 120                 125             


Pro Ile Tyr Pro Val Cys Ile Cys Pro Ala Gly Tyr Ala Gly Arg Phe 
    130                 135                 140                 


Cys Glu Ile Asp His Asp Glu Cys Ala Ser Ser Pro Cys Gln Asn Gly 
145                 150                 155                 160 


Ala Val Cys Gln Asp Gly Ile Asp Gly Tyr Ser Cys Phe Cys Val Pro 
                165                 170                 175     


Gly Tyr Gln Gly Arg His Cys Asp Leu Glu Val Asp Glu Cys Ala Ser 
            180                 185                 190         


Asp Pro Cys Lys Asn Glu Ala Thr Cys Leu Asn Glu Ile Gly Arg Tyr 
        195                 200                 205             


Thr Cys Ile Cys Pro His Asn Tyr Ser Gly Val Asn Cys Glu Leu Glu 
    210                 215                 220                 


Ile Asp Glu Cys Trp Ser Gln Pro Cys Leu Asn Gly Ala Thr Cys Gln 
225                 230                 235                 240 


Asp Ala Leu Gly Ala Tyr Phe Cys Asp Cys Ala Pro Gly Phe Leu Gly 
                245                 250                 255     


Asp His Cys Glu Leu Asn Thr Asp Glu Cys Ala Ser Gln Pro Cys Leu 
            260                 265                 270         


His Gly Gly Leu Cys Val Asp Gly Glu Asn Arg Tyr Ser Cys Asn Cys 
        275                 280                 285             


Thr Gly Ser Gly Phe Thr Gly Thr His Cys Glu Thr Leu Met Pro Leu 
    290                 295                 300                 


Cys Trp Ser Lys Pro Cys His Asn Asn Ala Thr Cys Glu Asp Ser Val 
305                 310                 315                 320 


Asp Asn Tyr Thr Cys His Cys Trp Pro Gly Tyr Thr Gly Ala Gln Cys 
                325                 330                 335     


Glu Ile Asp Leu Asn Glu Cys Asn Ser Asn Pro Cys Gln Ser Asn Gly 
            340                 345                 350         


Glu Cys Val Glu Leu Ser Ser Glu Lys Gln Tyr Gly Arg Ile Thr Gly 
        355                 360                 365             


Leu Pro Ser Ser Phe Ser Tyr His Glu Ala Ser Gly Tyr Val Cys Ile 
    370                 375                 380                 


Cys Gln Pro Gly Phe Thr Gly Ile His Cys Glu Glu Asp Val Asn Glu 
385                 390                 395                 400 


Cys Ser Ser Asn Pro Cys Gln Asn Gly Gly Thr Cys Glu Asn Leu Pro 
                405                 410                 415     


Gly Asn Tyr Thr Cys His Cys Pro Phe Asp Asn Leu Ser Arg Thr Phe 
            420                 425                 430         


Tyr Gly Gly Arg Asp Cys Ser Asp Ile Leu Leu Gly Cys Thr His Gln 
        435                 440                 445             


Gln Cys Leu Asn Asn Gly Thr Cys Ile Pro His Phe Gln Asp Gly Gln 
    450                 455                 460                 


His Gly Phe Ser Cys Leu Cys Pro Ser Gly Tyr Thr Gly Ser Leu Cys 
465                 470                 475                 480 


Glu Ile Ala Thr Thr Leu Ser Phe Glu Gly Asp Gly Phe Leu Trp Val 
                485                 490                 495     


Lys Ser Gly Ser Val Thr Thr Lys Gly Ser Val Cys Asn Ile Ala Leu 
            500                 505                 510         


Arg Phe Gln Thr Val Gln Pro Met Ala Leu Leu Leu Phe Arg Ser Asn 
        515                 520                 525             


Arg Asp Val Phe Val Lys Leu Glu Leu Leu Ser Gly Tyr Ile His Leu 
    530                 535                 540                 


Ser Ile Gln Val Asn Asn Gln Ser Lys Val Leu Leu Phe Ile Ser His 
545                 550                 555                 560 


Asn Thr Ser Asp Gly Glu Trp His Phe Val Glu Val Ile Phe Ala Glu 
                565                 570                 575     


Ala Val Thr Leu Thr Leu Ile Asp Asp Ser Cys Lys Glu Lys Cys Ile 
            580                 585                 590         


Ala Lys Ala Pro Thr Pro Leu Glu Ser Asp Gln Ser Ile Cys Ala Phe 
        595                 600                 605             


Gln Asn Ser Phe Leu Gly Gly Leu Pro Val Gly Met Thr Ser Asn Gly 
    610                 615                 620                 


Val Ala Leu Leu Asn Phe Tyr Asn Met Pro Ser Thr Pro Ser Phe Val 
625                 630                 635                 640 


Gly Cys Leu Gln Asp Ile Lys Ile Asp Trp Asn His Ile Thr Leu Glu 
                645                 650                 655     


Asn Ile Ser Ser Gly Ser Ser Leu Asn Val Lys Ala Gly Cys Val Arg 
            660                 665                 670         


Lys Asp Trp Cys Glu Ser Gln Pro Cys Gln Ser Arg Gly Arg Cys Ile 
        675                 680                 685             


Asn Leu Trp Leu Ser Tyr Gln Cys Asp Cys His Arg Pro Tyr Glu Gly 
    690                 695                 700                 


Pro Asn Cys Leu Arg Glu Tyr Val Ala Gly Arg Phe Gly Gln Asp Asp 
705                 710                 715                 720 


Ser Thr Gly Tyr Val Ile Phe Thr Leu Asp Glu Ser Tyr Gly Asp Thr 
                725                 730                 735     


Ile Ser Leu Ser Met Phe Val Arg Thr Leu Gln Pro Ser Gly Leu Leu 
            740                 745                 750         


Leu Ala Leu Glu Asn Ser Thr Tyr Gln Tyr Ile Arg Val Trp Leu Glu 
        755                 760                 765             


Arg Gly Arg Leu Ala Met Leu Thr Pro Asn Ser Pro Lys Leu Val Val 
    770                 775                 780                 


Lys Phe Val Leu Asn Asp Gly Asn Val His Leu Ile Ser Leu Lys Ile 
785                 790                 795                 800 


Lys Pro Tyr Lys Ile Glu Leu Tyr Gln Ser Ser Gln Asn Leu Gly Phe 
                805                 810                 815     


Ile Ser Ala Ser Thr Trp Lys Ile Glu Lys Gly Asp Val Ile Tyr Ile 
            820                 825                 830         


Gly Gly Leu Pro Asp Lys Gln Glu Thr Glu Leu Asn Gly Gly Phe Phe 
        835                 840                 845             


Lys Gly Cys Ile Gln Asp Val Arg Leu Asn Asn Gln Asn Leu Glu Phe 
    850                 855                 860                 


Phe Pro Asn Pro Thr Asn Asn Ala Ser Leu Asn Pro Val Leu Val Asn 
865                 870                 875                 880 


Val Thr Gln Gly Cys Ala Gly Asp Asn Ser Cys Lys Ser Asn Pro Cys 
                885                 890                 895     


His Asn Gly Gly Val Cys His Ser Arg Trp Asp Asp Phe Ser Cys Ser 
            900                 905                 910         


Cys Pro Ala Leu Thr Ser Gly Lys Ala Cys Glu Glu Val Gln Trp Cys 
        915                 920                 925             


Gly Phe Ser Pro Cys Pro His Gly Ala Gln Cys Gln Pro Val Leu Gln 
    930                 935                 940                 


Gly Phe Glu Cys Ile Ala Asn Ala Val Phe Asn Gly Gln Ser Gly Gln 
945                 950                 955                 960 


Ile Leu Phe Arg Ser Asn Gly Asn Ile Thr Arg Glu Leu Thr Asn Ile 
                965                 970                 975     


Thr Phe Gly Phe Arg Thr Arg Asp Ala Asn Val Ile Ile Leu His Ala 
            980                 985                 990         


Glu Lys Glu Pro Glu Phe Leu Asn  Ile Ser Ile Gln Asp  Ser Arg Leu 
        995                 1000                 1005             


Phe Phe  Gln Leu Gln Ser Gly  Asn Ser Phe Tyr Met  Leu Ser Leu 
    1010                 1015                 1020             


Thr Ser  Leu Gln Ser Val Asn  Asp Gly Thr Trp His  Glu Val Thr 
    1025                 1030                 1035             


Leu Ser  Met Thr Asp Pro Leu  Ser Gln Thr Ser Arg  Trp Gln Met 
    1040                 1045                 1050             


Glu Val  Asp Asn Glu Thr Pro  Phe Val Thr Ser Thr  Ile Ala Thr 
    1055                 1060                 1065             


Gly Ser  Leu Asn Phe Leu Lys  Asp Asn Thr Asp Ile  Tyr Val Gly 
    1070                 1075                 1080             


Asp Arg  Ala Ile Asp Asn Ile  Lys Gly Leu Gln Gly  Cys Leu Ser 
    1085                 1090                 1095             


Thr Ile  Glu Ile Gly Gly Ile  Tyr Leu Ser Tyr Phe  Glu Asn Val 
    1100                 1105                 1110             


His Gly  Phe Ile Asn Lys Pro  Gln Glu Glu Gln Phe  Leu Lys Ile 
    1115                 1120                 1125             


Ser Thr  Asn Ser Val Val Thr  Gly Cys Leu Gln Leu  Asn Val Cys 
    1130                 1135                 1140             


Asn Ser  Asn Pro Cys Leu His  Gly Gly Asn Cys Glu  Asp Ile Tyr 
    1145                 1150                 1155             


Ser Ser  Tyr His Cys Ser Cys  Pro Leu Gly Trp Ser  Gly Lys His 
    1160                 1165                 1170             


Cys Glu  Leu Asn Ile Asp Glu  Cys Phe Ser Asn Pro  Cys Ile His 
    1175                 1180                 1185             


Gly Asn  Cys Ser Asp Arg Val  Ala Ala Tyr His Cys  Thr Cys Glu 
    1190                 1195                 1200             


Pro Gly  Tyr Thr Gly Val Asn  Cys Glu Val Asp Ile  Asp Asn Cys 
    1205                 1210                 1215             


Gln Ser  His Gln Cys Ala Asn  Gly Ala Thr Cys Ile  Ser His Thr 
    1220                 1225                 1230             


Asn Gly  Tyr Ser Cys Leu Cys  Phe Gly Asn Phe Thr  Gly Lys Phe 
    1235                 1240                 1245             


Cys Arg  Gln Ser Arg Leu Pro  Ser Thr Val Cys Gly  Asn Glu Lys 
    1250                 1255                 1260             


Thr Asn  Leu Thr Cys Tyr Asn  Gly Gly Asn Cys Thr  Glu Phe Gln 
    1265                 1270                 1275             


Thr Glu  Leu Lys Cys Met Cys  Arg Pro Gly Phe Thr  Gly Glu Trp 
    1280                 1285                 1290             


Cys Glu  Lys Asp Ile Asp Glu  Cys Ala Ser Asp Pro  Cys Val Asn 
    1295                 1300                 1305             


Gly Gly  Leu Cys Gln Asp Leu  Leu Asn Lys Phe Gln  Cys Leu Cys 
    1310                 1315                 1320             


Asp Val  Ala Phe Ala Gly Glu  Arg Cys Glu Val Asp  Leu Ala Asp 
    1325                 1330                 1335             


Asp Leu  Ile Ser Asp Ile Phe  Thr Thr Ile Gly Ser  Val Thr Val 
    1340                 1345                 1350             


Ala Leu  Leu Leu Ile Leu Leu  Leu Ala Ile Val Ala  Ser Val Val 
    1355                 1360                 1365             


Thr Ser  Asn Lys Arg Ala Thr  Gln Gly Thr Tyr Ser  Pro Ser Arg 
    1370                 1375                 1380             


Gln Glu  Lys Glu Gly Ser Arg  Val Glu Met Trp Asn  Leu Met Pro 
    1385                 1390                 1395             


Pro Pro  Ala Met Glu Arg Leu  Ile 
    1400                 1405     


<210>  9
<211>  1003
<212>  PRT
<213>  Homo sapiens

<400>  9

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


Ile Gly Ile His Cys Glu Glu Asp Val Asn Glu Cys Ser Ser Asn Pro 
            20                  25                  30          


Cys Gln Asn Gly Gly Thr Cys Glu Asn Leu Pro Gly Asn Tyr Thr Cys 
        35                  40                  45              


His Cys Pro Phe Asp Asn Leu Ser Arg Thr Phe Tyr Gly Gly Arg Asp 
    50                  55                  60                  


Cys Ser Asp Ile Leu Leu Gly Cys Thr His Gln Gln Cys Leu Asn Asn 
65                  70                  75                  80  


Gly Thr Cys Ile Pro His Phe Gln Asp Gly Gln His Gly Phe Ser Cys 
                85                  90                  95      


Leu Cys Pro Ser Gly Tyr Thr Gly Ser Leu Cys Glu Ile Ala Thr Thr 
            100                 105                 110         


Leu Ser Phe Glu Gly Asp Gly Phe Leu Trp Val Lys Ser Gly Ser Val 
        115                 120                 125             


Thr Thr Lys Gly Ser Val Cys Asn Ile Ala Leu Arg Phe Gln Thr Val 
    130                 135                 140                 


Gln Pro Met Ala Leu Leu Leu Phe Arg Ser Asn Arg Asp Val Phe Val 
145                 150                 155                 160 


Lys Leu Glu Leu Leu Ser Gly Tyr Ile His Leu Ser Ile Gln Val Asn 
                165                 170                 175     


Asn Gln Ser Lys Val Leu Leu Phe Ile Ser His Asn Thr Ser Asp Gly 
            180                 185                 190         


Glu Trp His Phe Val Glu Val Ile Phe Ala Glu Ala Val Thr Leu Thr 
        195                 200                 205             


Leu Ile Asp Asp Ser Cys Lys Glu Lys Cys Ile Ala Lys Ala Pro Thr 
    210                 215                 220                 


Pro Leu Glu Ser Asp Gln Ser Ile Cys Ala Phe Gln Asn Ser Phe Leu 
225                 230                 235                 240 


Gly Gly Leu Pro Val Gly Met Thr Ser Asn Gly Val Ala Leu Leu Asn 
                245                 250                 255     


Phe Tyr Asn Met Pro Ser Thr Pro Ser Phe Val Gly Cys Leu Gln Asp 
            260                 265                 270         


Ile Lys Ile Asp Trp Asn His Ile Thr Leu Glu Asn Ile Ser Ser Gly 
        275                 280                 285             


Ser Ser Leu Asn Val Lys Ala Gly Cys Val Arg Lys Asp Trp Cys Glu 
    290                 295                 300                 


Ser Gln Pro Cys Gln Ser Arg Gly Arg Cys Ile Asn Leu Trp Leu Ser 
305                 310                 315                 320 


Tyr Gln Cys Asp Cys His Arg Pro Tyr Glu Gly Pro Asn Cys Leu Arg 
                325                 330                 335     


Glu Tyr Val Ala Gly Arg Phe Gly Gln Asp Asp Ser Thr Gly Tyr Val 
            340                 345                 350         


Ile Phe Thr Leu Asp Glu Ser Tyr Gly Asp Thr Ile Ser Leu Ser Met 
        355                 360                 365             


Phe Val Arg Thr Leu Gln Pro Ser Gly Leu Leu Leu Ala Leu Glu Asn 
    370                 375                 380                 


Ser Thr Tyr Gln Tyr Ile Arg Val Trp Leu Glu Arg Gly Arg Leu Ala 
385                 390                 395                 400 


Met Leu Thr Pro Asn Ser Pro Lys Leu Val Val Lys Phe Val Leu Asn 
                405                 410                 415     


Asp Gly Asn Val His Leu Ile Ser Leu Lys Ile Lys Pro Tyr Lys Ile 
            420                 425                 430         


Glu Leu Tyr Gln Ser Ser Gln Asn Leu Gly Phe Ile Ser Ala Ser Thr 
        435                 440                 445             


Trp Lys Ile Glu Lys Gly Asp Val Ile Tyr Ile Gly Gly Leu Pro Asp 
    450                 455                 460                 


Lys Gln Glu Thr Glu Leu Asn Gly Gly Phe Phe Lys Gly Cys Ile Gln 
465                 470                 475                 480 


Asp Val Arg Leu Asn Asn Gln Asn Leu Glu Phe Phe Pro Asn Pro Thr 
                485                 490                 495     


Asn Asn Ala Ser Leu Asn Pro Val Leu Val Asn Val Thr Gln Gly Cys 
            500                 505                 510         


Ala Gly Asp Asn Ser Cys Lys Ser Asn Pro Cys His Asn Gly Gly Val 
        515                 520                 525             


Cys His Ser Arg Trp Asp Asp Phe Ser Cys Ser Cys Pro Ala Leu Thr 
    530                 535                 540                 


Ser Gly Lys Ala Cys Glu Glu Val Gln Trp Cys Gly Phe Ser Pro Cys 
545                 550                 555                 560 


Pro His Gly Ala Gln Cys Gln Pro Val Leu Gln Gly Phe Glu Cys Ile 
                565                 570                 575     


Ala Asn Ala Val Phe Asn Gly Gln Ser Gly Gln Ile Leu Phe Arg Ser 
            580                 585                 590         


Asn Gly Asn Ile Thr Arg Glu Leu Thr Asn Ile Thr Phe Gly Phe Arg 
        595                 600                 605             


Thr Arg Asp Ala Asn Val Ile Ile Leu His Ala Glu Lys Glu Pro Glu 
    610                 615                 620                 


Phe Leu Asn Ile Ser Ile Gln Asp Ser Arg Leu Phe Phe Gln Leu Gln 
625                 630                 635                 640 


Ser Gly Asn Ser Phe Tyr Met Leu Ser Leu Thr Ser Leu Gln Ser Val 
                645                 650                 655     


Asn Asp Gly Thr Trp His Glu Val Thr Leu Ser Met Thr Asp Pro Leu 
            660                 665                 670         


Ser Gln Thr Ser Arg Trp Gln Met Glu Val Asp Asn Glu Thr Pro Phe 
        675                 680                 685             


Val Thr Ser Thr Ile Ala Thr Gly Ser Leu Asn Phe Leu Lys Asp Asn 
    690                 695                 700                 


Thr Asp Ile Tyr Val Gly Asp Arg Ala Ile Asp Asn Ile Lys Gly Leu 
705                 710                 715                 720 


Gln Gly Cys Leu Ser Thr Ile Glu Ile Gly Gly Ile Tyr Leu Ser Tyr 
                725                 730                 735     


Phe Glu Asn Val His Gly Phe Ile Asn Lys Pro Gln Glu Glu Gln Phe 
            740                 745                 750         


Leu Lys Ile Ser Thr Asn Ser Val Val Thr Gly Cys Leu Gln Leu Asn 
        755                 760                 765             


Val Cys Asn Ser Asn Pro Cys Leu His Gly Gly Asn Cys Glu Asp Ile 
    770                 775                 780                 


Tyr Ser Ser Tyr His Cys Ser Cys Pro Leu Gly Trp Ser Gly Lys His 
785                 790                 795                 800 


Cys Glu Leu Asn Ile Asp Glu Cys Phe Ser Asn Pro Cys Ile His Gly 
                805                 810                 815     


Asn Cys Ser Asp Arg Val Ala Ala Tyr His Cys Thr Cys Glu Pro Gly 
            820                 825                 830         


Tyr Thr Gly Val Asn Cys Glu Val Asp Ile Asp Asn Cys Gln Ser His 
        835                 840                 845             


Gln Cys Ala Asn Gly Ala Thr Cys Ile Ser His Thr Asn Gly Tyr Ser 
    850                 855                 860                 


Cys Leu Cys Phe Gly Asn Phe Thr Gly Lys Phe Cys Arg Gln Ser Arg 
865                 870                 875                 880 


Leu Pro Ser Thr Val Cys Gly Asn Glu Lys Thr Asn Leu Thr Cys Tyr 
                885                 890                 895     


Asn Gly Gly Asn Cys Thr Glu Phe Gln Thr Glu Leu Lys Cys Met Cys 
            900                 905                 910         


Arg Pro Gly Phe Thr Gly Glu Trp Cys Glu Lys Asp Ile Asp Glu Cys 
        915                 920                 925             


Ala Ser Asp Pro Cys Val Asn Gly Gly Leu Cys Gln Asp Leu Leu Asn 
    930                 935                 940                 


Lys Phe Gln Cys Leu Cys Asp Val Ala Phe Ala Gly Glu Arg Cys Glu 
945                 950                 955                 960 


Val Asp Val Ser Ser Leu Ser Phe Tyr Val Ser Leu Leu Phe Trp Gln 
                965                 970                 975     


Asn Leu Phe Gln Leu Leu Ser Tyr Leu Ile Leu Arg Met Asn Asp Glu 
            980                 985                 990         


Pro Val Val Glu Trp Gly Glu Gln  Glu Asp Tyr 
        995                 1000             


