                         SEQUENCE LISTING

<110>  ALBERT EINSTEIN COLLEGE OF MEDICINE OF YESHIVA UNIVERSITY
 
<120>  PAK1 INHIBITION FOR TREATMENT OF ACUTE MYELOID LEUKEMIA AND 
       MYELODYSPLASTIC SYNDROMES

<130>  96700/2106

<140>  PCT/US2014/24239
<141>  2014-03-12

<150>  US 61/789,345
<151>  2013-03-15

<160>  35    

<170>  PatentIn version 3.5

<210>  1
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse H1x forward primer

<400>  1
ttcagcatca attccaagac aca                                               23


<210>  2
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse H1x reverse primer

<400>  2
acctcttctc caggcctttt ct                                                22


<210>  3
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse Btgl1 foward primer

<400>  3
tcatctccaa gttcctccgc ac                                                22


<210>  4
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse Btgl1 reverse primer

<400>  4
caacggtaac ctgatccctt gc                                                22


<210>  5
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse FoxO4 forward primer

<400>  5
tctacgaatg gatggtccgc ac                                                22


<210>  6
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse FoxO4 reverse primer

<400>  6
cttgctgtgc aaggacaggt tg                                                22


<210>  7
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse Gadd45a forward primer

<400>  7
cctggaggaa gtgctcagca ag                                                22


<210>  8
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse Gadd45a reverse primer

<400>  8
gtcgtcttcg tcagcagcca g                                                 21


<210>  9
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse Hdac7 forward primer

<400>  9
cgcctcaaac tggataacgg ga                                                22


<210>  10
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse Hdac7 reverse primer

<400>  10
gcattggagg aatgcagctc gt                                                22


<210>  11
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse Pak1 foward primer

<400>  11
attgctccac gcccagaaca ca                                                22


<210>  12
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse Pak1 reverse primer

<400>  12
aagcatctgg cggagtggtg tt                                                22


<210>  13
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse Gapdh foward primer

<400>  13
ccagcctcgt cccgtagac                                                    19


<210>  14
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse Gapdh reverse primer

<400>  14
gccttgactg tgccgttga                                                    19


<210>  15
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse Trp63 forward primer

<400>  15
tgagccgtga gttcaatgag                                                   20


<210>  16
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse Trp63 reverse primer

<400>  16
acctgtggtg gctcataagg                                                   20


<210>  17
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse RhoB forward primer

<400>  17
tattttcgga ggcagaatgg                                                   20


<210>  18
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse RhoB reverse primer

<400>  18
gatggccaag tccagttcat                                                   20


<210>  19
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse Zfp361 forward primer

<400>  19
cccctacctt ggacaactca                                                   20


<210>  20
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse Zfp361 reverse primer

<400>  20
aagagatggg gtggagaggt                                                   20


<210>  21
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse Fyn forward primer

<400>  21
ggaaaaagga tccggaagag                                                   20


<210>  22
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  mouse Fyn reverse primer

<400>  22
gcaggctctc acaggttttc                                                   20


<210>  23
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  human PAK1 forward primer

<400>  23
gtgaaggctg tgtctgagac tc                                                22


<210>  24
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  human PAK1 reverse primer

<400>  24
ggaagtggtt caatcacaga ccg                                               23


<210>  25
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  human BTG1 forward primer

<400>  25
catctccaag tttctccgca cc                                                22


<210>  26
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  human BTG1 reverse primer

<400>  26
gcgaatacaa cggtaacccg atc                                               23


<210>  27
<211>  26
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  short hairpin sequence for luciferase target

<400>  27
gtgcgttgtt agtactaatc ctattt                                            26


<210>  28
<211>  29
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  short hairpin sequence for mouse H1x target

<400>  28
ggcgcagaag gacaaggaca aggaagcgg                                         29


<210>  29
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  non-silencing control

<400>  29
acctccaccc tcactctgcc at                                                22


<210>  30
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  short hairpin sequence for human HLX target

<400>  30
ccctatgctg tgctcacga                                                    19


<210>  31
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  short hairpin sequence for human PAK1 #1

<400>  31
cgatgagaaa taccagcact a                                                 21


<210>  32
<211>  21
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  short hairpin sequence for human PAK1 #2

<400>  32
gcgatcctaa gaagaaatat a                                                 21


<210>  33
<211>  2308
<212>  DNA
<213>  Homo sapiens

<400>  33
aaaactttgg gagtttttag agacgagttt tttttttttt ctattacttt tccccccccc       60

taactaacgg actattattg ttgttgtttt aaatttagct cttagggctt agctatttgg      120

gttttcttgc ggtgtccggc tcccgtctcc ctggctcccc cgcccgccct gcggccccag      180

cgcccctcgc tctcatccag cccgcgagga gtgcgggcgc cgcgccgcct ttaaagcgag      240

gccagggagc gaggcggtga ccggccgaga tccggccctc gcctcctccc tcggtggcgc      300

tagggctccc ggcctctctt cctcagtgcg ggcggagaag cgaaagcgga tcgtcctcgg      360

ctgccgccgc cttctccggg actcgcgcgc ccctccccgc gcgcccaccc acccagtccg      420

gctggactgc ggcagccgcg cggctcaccc cggcaggatg ttcgcagccg ggctggctcc      480

cttctacgcc tccaacttca gcctctggtc ggccgcttac tgctcctcgg ccggcccagg      540

cggctgctcc ttccccttgg accccgccgc cgtcaaaaag ccctccttct gcatcgcaga      600

cattctgcac gccggcgtgg gggatctggg ggcggccccg gagggcctgg caggggcctc      660

ggccgccgcc ctcaccgcgc acttgggctc ggttcacccg cacgcctctt tccaagcggc      720

ggccagatcc ccgcttcgac ccaccccagt ggtggcgccc tccgaagtcc cggctggctt      780

cccgcagcgg ctgtctccgc tctcagccgc ctaccaccac catcacccgc aacaacaaca      840

gcagcagcaa cagccgcagc agcaacagcc tccgcctccg ccccgggctg gcgccctgca      900

gcccccggcc tcggggacgc gagtggttcc gaacccccac cacagtggct ctgccccggc      960

cccctccagc aaagacctca aatttggaat tgaccgcatt ttatctgcag aatttgaccc     1020

aaaagtcaaa gaaggcaaca cgctgagaga tctcacttcc ctgctaaccg gtgggcggcc     1080

cgccggggtg cacctctcag gcctgcagcc ctcggccggc cagttcttcg catctctaga     1140

tcccattaac gaggcttctg caatcctgag tcccttaaac tcgaacccaa gaaattcagt     1200

tcagcatcag ttccaagaca cgtttccagg tccctatgct gtgctcacga aggacaccat     1260

gccgcagacg tacaaaagga agcgttcatg gtcgcgcgct gtgttctcca acctgcagag     1320

gaaaggcctg gagaaaaggt ttgagattca gaagtacgtg accaagccgg accgaaagca     1380

gctggcggcg atgctgggcc tcacggacgc acaggtgaag gtgtggttcc agaaccggcg     1440

gatgaagtgg cggcactcca aggaggccca ggcccaaaag gacaaggaca aggaggctgg     1500

cgagaagcca tcaggtggag ccccggctgc ggatggcgag caggacgaga ggagccccag     1560

ccgttctgaa ggcgaggctg agagcgagag cagcgactcc gagtccctgg acatggcccc     1620

cagcgacacg gagcggactg aggggagtga gcgttctctg caccaaacaa cagttattaa     1680

ggccccggtc actggcgccc tcattaccgc cagcagtgct gggagtggtg ggagcagcgg     1740

cggcggcggc aatagtttca gcttcagcag cgccagcagt cttagtagca gcagcaccag     1800

tgcgggttgc gccagcagcc ttggcggcgg cggcgcctcg gagcttctcc ctgcaacaca     1860

gcccacagcc agcagcgctc ccaaaagccc cgagccagcc caaggcgcgc ttggctgctt     1920

atagactgta ctagggcgga ggggatccgg gccttgcgtg cagcctccca accatgggct     1980

gggttttgtg cttactgtat gttggcgact tggtagggca ggagacgcag cgtggagcct     2040

acctcccgac attcacgctt cgccccacgc tgctccgact ggctgcagcg gacactgccc     2100

aaagcagagg ggagtctcag tgtcctgcta gccagccgaa cacttctctc cggaagcagg     2160

ctggttcgac tgtgaggtgt ttgactaaac tgtttctctg actcgcccca gaggtcgtgg     2220

ctcaaaggca cttaggacgc cttaaatttg taaataaaat gtttactacg gtttgtaaaa     2280

aaaaaaaaaa aaaaaaaaaa aaaaaaaa                                        2308


<210>  34
<211>  488
<212>  PRT
<213>  Homo sapiens

<400>  34

Met Phe Ala Ala Gly Leu Ala Pro Phe Tyr Ala Ser Asn Phe Ser Leu 
1               5                   10                  15      


Trp Ser Ala Ala Tyr Cys Ser Ser Ala Gly Pro Gly Gly Cys Ser Phe 
            20                  25                  30          


Pro Leu Asp Pro Ala Ala Val Lys Lys Pro Ser Phe Cys Ile Ala Asp 
        35                  40                  45              


Ile Leu His Ala Gly Val Gly Asp Leu Gly Ala Ala Pro Glu Gly Leu 
    50                  55                  60                  


Ala Gly Ala Ser Ala Ala Ala Leu Thr Ala His Leu Gly Ser Val His 
65                  70                  75                  80  


Pro His Ala Ser Phe Gln Ala Ala Ala Arg Ser Pro Leu Arg Pro Thr 
                85                  90                  95      


Pro Val Val Ala Pro Ser Glu Val Pro Ala Gly Phe Pro Gln Arg Leu 
            100                 105                 110         


Ser Pro Leu Ser Ala Ala Tyr His His His His Pro Gln Gln Gln Gln 
        115                 120                 125             


Gln Gln Gln Gln Pro Gln Gln Gln Gln Pro Pro Pro Pro Pro Arg Ala 
    130                 135                 140                 


Gly Ala Leu Gln Pro Pro Ala Ser Gly Thr Arg Val Val Pro Asn Pro 
145                 150                 155                 160 


His His Ser Gly Ser Ala Pro Ala Pro Ser Ser Lys Asp Leu Lys Phe 
                165                 170                 175     


Gly Ile Asp Arg Ile Leu Ser Ala Glu Phe Asp Pro Lys Val Lys Glu 
            180                 185                 190         


Gly Asn Thr Leu Arg Asp Leu Thr Ser Leu Leu Thr Gly Gly Arg Pro 
        195                 200                 205             


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


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


Asn Ser Asn Pro Arg Asn Ser Val Gln His Gln Phe Gln Asp Thr Phe 
                245                 250                 255     


Pro Gly Pro Tyr Ala Val Leu Thr Lys Asp Thr Met Pro Gln Thr Tyr 
            260                 265                 270         


Lys Arg Lys Arg Ser Trp Ser Arg Ala Val Phe Ser Asn Leu Gln Arg 
        275                 280                 285             


Lys Gly Leu Glu Lys Arg Phe Glu Ile Gln Lys Tyr Val Thr Lys Pro 
    290                 295                 300                 


Asp Arg Lys Gln Leu Ala Ala Met Leu Gly Leu Thr Asp Ala Gln Val 
305                 310                 315                 320 


Lys Val Trp Phe Gln Asn Arg Arg Met Lys Trp Arg His Ser Lys Glu 
                325                 330                 335     


Ala Gln Ala Gln Lys Asp Lys Asp Lys Glu Ala Gly Glu Lys Pro Ser 
            340                 345                 350         


Gly Gly Ala Pro Ala Ala Asp Gly Glu Gln Asp Glu Arg Ser Pro Ser 
        355                 360                 365             


Arg Ser Glu Gly Glu Ala Glu Ser Glu Ser Ser Asp Ser Glu Ser Leu 
    370                 375                 380                 


Asp Met Ala Pro Ser Asp Thr Glu Arg Thr Glu Gly Ser Glu Arg Ser 
385                 390                 395                 400 


Leu His Gln Thr Thr Val Ile Lys Ala Pro Val Thr Gly Ala Leu Ile 
                405                 410                 415     


Thr Ala Ser Ser Ala Gly Ser Gly Gly Ser Ser Gly Gly Gly Gly Asn 
            420                 425                 430         


Ser Phe Ser Phe Ser Ser Ala Ser Ser Leu Ser Ser Ser Ser Thr Ser 
        435                 440                 445             


Ala Gly Cys Ala Ser Ser Leu Gly Gly Gly Gly Ala Ser Glu Leu Leu 
    450                 455                 460                 


Pro Ala Thr Gln Pro Thr Ala Ser Ser Ala Pro Lys Ser Pro Glu Pro 
465                 470                 475                 480 


Ala Gln Gly Ala Leu Gly Cys Leu 
                485             


<210>  35
<211>  545
<212>  PRT
<213>  Homo sapiens

<400>  35

Met Ser Asn Asn Gly Leu Asp Ile Gln Asp Lys Pro Pro Ala Pro Pro 
1               5                   10                  15      


Met Arg Asn Thr Ser Thr Met Ile Gly Ala Gly Ser Lys Asp Ala Gly 
            20                  25                  30          


Thr Leu Asn His Gly Ser Lys Pro Leu Pro Pro Asn Pro Glu Glu Lys 
        35                  40                  45              


Lys Lys Lys Asp Arg Phe Tyr Arg Ser Ile Leu Pro Gly Asp Lys Thr 
    50                  55                  60                  


Asn Lys Lys Lys Glu Lys Glu Arg Pro Glu Ile Ser Leu Pro Ser Asp 
65                  70                  75                  80  


Phe Glu His Thr Ile His Val Gly Phe Asp Ala Val Thr Gly Glu Phe 
                85                  90                  95      


Thr Gly Met Pro Glu Gln Trp Ala Arg Leu Leu Gln Thr Ser Asn Ile 
            100                 105                 110         


Thr Lys Ser Glu Gln Lys Lys Asn Pro Gln Ala Val Leu Asp Val Leu 
        115                 120                 125             


Glu Phe Tyr Asn Ser Lys Lys Thr Ser Asn Ser Gln Lys Tyr Met Ser 
    130                 135                 140                 


Phe Thr Asp Lys Ser Ala Glu Asp Tyr Asn Ser Ser Asn Ala Leu Asn 
145                 150                 155                 160 


Val Lys Ala Val Ser Glu Thr Pro Ala Val Pro Pro Val Ser Glu Asp 
                165                 170                 175     


Glu Asp Asp Asp Asp Asp Asp Ala Thr Pro Pro Pro Val Ile Ala Pro 
            180                 185                 190         


Arg Pro Glu His Thr Lys Ser Val Tyr Thr Arg Ser Val Ile Glu Pro 
        195                 200                 205             


Leu Pro Val Thr Pro Thr Arg Asp Val Ala Thr Ser Pro Ile Ser Pro 
    210                 215                 220                 


Thr Glu Asn Asn Thr Thr Pro Pro Asp Ala Leu Thr Arg Asn Thr Glu 
225                 230                 235                 240 


Lys Gln Lys Lys Lys Pro Lys Met Ser Asp Glu Glu Ile Leu Glu Lys 
                245                 250                 255     


Leu Arg Ser Ile Val Ser Val Gly Asp Pro Lys Lys Lys Tyr Thr Arg 
            260                 265                 270         


Phe Glu Lys Ile Gly Gln Gly Ala Ser Gly Thr Val Tyr Thr Ala Met 
        275                 280                 285             


Asp Val Ala Thr Gly Gln Glu Val Ala Ile Lys Gln Met Asn Leu Gln 
    290                 295                 300                 


Gln Gln Pro Lys Lys Glu Leu Ile Ile Asn Glu Ile Leu Val Met Arg 
305                 310                 315                 320 


Glu Asn Lys Asn Pro Asn Ile Val Asn Tyr Leu Asp Ser Tyr Leu Val 
                325                 330                 335     


Gly Asp Glu Leu Trp Val Val Met Glu Tyr Leu Ala Gly Gly Ser Leu 
            340                 345                 350         


Thr Asp Val Val Thr Glu Thr Cys Met Asp Glu Gly Gln Ile Ala Ala 
        355                 360                 365             


Val Cys Arg Glu Cys Leu Gln Ala Leu Glu Phe Leu His Ser Asn Gln 
    370                 375                 380                 


Val Ile His Arg Asp Ile Lys Ser Asp Asn Ile Leu Leu Gly Met Asp 
385                 390                 395                 400 


Gly Ser Val Lys Leu Thr Asp Phe Gly Phe Cys Ala Gln Ile Thr Pro 
                405                 410                 415     


Glu Gln Ser Lys Arg Ser Thr Met Val Gly Thr Pro Tyr Trp Met Ala 
            420                 425                 430         


Pro Glu Val Val Thr Arg Lys Ala Tyr Gly Pro Lys Val Asp Ile Trp 
        435                 440                 445             


Ser Leu Gly Ile Met Ala Ile Glu Met Ile Glu Gly Glu Pro Pro Tyr 
    450                 455                 460                 


Leu Asn Glu Asn Pro Leu Arg Ala Leu Tyr Leu Ile Ala Thr Asn Gly 
465                 470                 475                 480 


Thr Pro Glu Leu Gln Asn Pro Glu Lys Leu Ser Ala Ile Phe Arg Asp 
                485                 490                 495     


Phe Leu Asn Arg Cys Leu Glu Met Asp Val Glu Lys Arg Gly Ser Ala 
            500                 505                 510         


Lys Glu Leu Leu Gln His Gln Phe Leu Lys Ile Ala Lys Pro Leu Ser 
        515                 520                 525             


Ser Leu Thr Pro Leu Ile Ala Ala Ala Lys Glu Ala Thr Lys Asn Asn 
    530                 535                 540                 


His 
545 


