                         SEQUENCE LISTING

<110>  National Human Genome Research Institute
       University of Maryland
 
<120>  METHODS FOR TREATING PROGEROID LAMINOPATHIES USING 
       OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA

<130>  120178.49401WO

<150>  US 61/568,590
<151>  2011-12-08

<160>  54    

<170>  PatentIn version 3.5

<210>  1
<211>  270
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary LMNA Target Sequence

<400>  1
ggctcccact gcagcagctc gggggacccc gctgagtaca acctgcgctc gcgcaccgtg       60

ctgtgcggga cctgcgggca gcctgccgac aaggcatctg ccagcggctc aggagcccag      120

gtgggcggac ccatctcctc tggctcttct gcctccagtg tcacggtcac tcgcagctac      180

cgcagtgtgg ggggcagtgg gggtggcagc ttcggggaca atctggtcac ccgctcctac      240

ctcctgggca actccagccc ccgaacccag                                       270


<210>  2
<211>  270
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary LMNA Target Sequence

<400>  2
ggctcccact gcagcagctc gggggacccc gctgagtaca acctgcgctc gcgcaccgtg       60

ctgtgcggga cctgcgggca gcctgccgac aaggcatctg ccagcggctc aggagcccag      120

gtgggtggac ccatctcctc tggctcttct gcctccagtg tcacggtcac tcgcagctac      180

cgcagtgtgg ggggcagtgg gggtggcagc ttcggggaca atctggtcac ccgctcctac      240

ctcctgggca actccagccc ccgaacccag                                       270


<210>  3
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  3
ccgctggcag atgccttgtc ggcag                                             25


<210>  4
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  4
ctgagccgct ggcagatgcc ttgtc                                             25


<210>  5
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  5
gctcctgagc cgctggcaga tgcct                                             25


<210>  6
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  6
tgggctcctg agccgctggc agatg                                             25


<210>  7
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequences

<400>  7
cacctgggct cctgagccgc tggca                                             25


<210>  8
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  8
ccacccacct gggctcctga gccgc                                             25


<210>  9
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  9
gggtccaccc acctgggctc ctgag                                             25


<210>  10
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  10
agatgggtcc acccacctgg gctcc                                             25


<210>  11
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  11
gaggagatgg gtccacccac ctggg                                             25


<210>  12
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  12
gccagaggag atgggtccac ccacc                                             25


<210>  13
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  13
aagagccaga ggagatgggt ccacc                                             25


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

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  14
cagaagagcc agaggagatg ggtcc                                             25


<210>  15
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  15
gaggcagaag agccagagga gatgg                                             25


<210>  16
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  16
actggaggca gaagagccag aggag                                             25


<210>  17
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  17
acgtggtggt gatggagcag gtcat                                             25


<210>  18
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  18
actcacgtgg tggtgatgga gcagg                                             25


<210>  19
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  19
gctaccactc acgtggtggt gatgg                                             25


<210>  20
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  20
cggcggctac cactcacgtg gtggt                                             25


<210>  21
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  21
cagcggcggc taccactcac gtggt                                             25


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

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  22
cctcagcggc ggctaccact cacgt                                             25


<210>  23
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  23
ggcctcagcg gcggctacca ctcac                                             25


<210>  24
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  24
gctcggcctc agcggcggct accac                                             25


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

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  25
cgagtctggg actgaccact caggc                                             25


<210>  26
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  26
aggctcaggc gggacggcga gtctg                                             25


<210>  27
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  27
agacaaggct caggcgggac ggcga                                             25


<210>  28
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  28
agggagacaa ggctcaggcg ggacg                                             25


<210>  29
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  29
gggaagggag acaaggctca ggcgg                                             25


<210>  30
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  30
gccctgggaa gggagacaag gctca                                             25


<210>  31
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  31
gtgggagccc tgggaaggga gacaa                                             25


<210>  32
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  32
ctgctgcagt gggagccctg ggaag                                             25


<210>  33
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  33
agctgctgca gtgggagccc tggga                                             25


<210>  34
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence

<400>  34
cccccgagct gctgcagtgg gagcc                                             25


<210>  35
<211>  26
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence


<220>
<221>  misc_feature
<222>  (26)..(26)
<223>  n = a preferred cell penetrating peptide transporter

<400>  35
gctaccactc acgtggtggt gatggn                                            26


<210>  36
<211>  26
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence


<220>
<221>  misc_feature
<222>  (26)..(26)
<223>  n = a preferred cell-penetrating peptide transporter

<400>  36
gggtccaccc acctgggctc ctgagn                                            26


<210>  37
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence


<220>
<221>  misc_feature
<222>  (3)..(3)
<223>  n = apn intersubunit linkage

<220>
<221>  misc_feature
<222>  (9)..(9)
<223>  n = apn intersubunit linkage

<220>
<221>  misc_feature
<222>  (14)..(14)
<223>  n = apn intersubunit linkage

<220>
<221>  misc_feature
<222>  (20)..(20)
<223>  n = apn intersubunit linkage

<400>  37
gcnaccacnc acgnggtggn gatgg                                             25


<210>  38
<211>  25
<212>  DNA
<213>  Artificial

<220>
<223>  Exemplary HGPS Targeting Sequence


<220>
<221>  misc_feature
<222>  (4)..(4)
<223>  n = apn intersubunit linkage

<220>
<221>  misc_feature
<222>  (14)..(14)
<223>  n = apn intersubunit linkage

<220>
<221>  misc_feature
<222>  (19)..(19)
<223>  n = apn intersubunit linkage

<220>
<221>  misc_feature
<222>  (22)..(22)
<223>  n = apn intersubunit linkage

<400>  38
gggnccaccc accngggcnc cngag                                             25


<210>  39
<211>  9
<212>  PRT
<213>  Artificial

<220>
<223>  Arginine-Rich Cell-Penetrating Peptide Transporter

<400>  39

Arg Arg Arg Gln Arg Arg Lys Lys Arg 
1               5                   


<210>  40
<211>  9
<212>  PRT
<213>  Artificial

<220>
<223>  Arginine-Rich Cell-Penetrating Peptide Transporter

<400>  40

Arg Lys Lys Arg Arg Gln Arg Arg Arg 
1               5                   


<210>  41
<211>  11
<212>  PRT
<213>  Artificial

<220>
<223>  Arginine-Rich Cell-Penetrating Peptide Transporter

<400>  41

Arg Arg Arg Arg Arg Arg Arg Arg Arg Phe Phe 
1               5                   10      


<210>  42
<211>  11
<212>  PRT
<213>  Artificial

<220>
<223>  Arginine-Rich Cell-Penetrating Peptide Transporter

<400>  42

Arg Arg Arg Arg Arg Phe Phe Arg Arg Arg Arg 
1               5                   10      


<210>  43
<211>  4
<212>  PRT
<213>  Artificial

<220>
<223>  Arginine-Rich Cell-Penetrating Peptide Transporters

<400>  43

Arg Arg Arg Arg 
1               


<210>  44
<211>  5
<212>  PRT
<213>  Artificial

<220>
<223>  Arginine-Rich Cell-Penetrating Peptide Transporter

<400>  44

Arg Arg Arg Arg Arg 
1               5   


<210>  45
<211>  6
<212>  PRT
<213>  Artificial

<220>
<223>  Arginine-Rich Cell-Penetrating Peptide Transporter

<400>  45

Arg Arg Arg Arg Arg Arg 
1               5       


<210>  46
<211>  7
<212>  PRT
<213>  Artificial

<220>
<223>  Arginine-Rich Cell-Penetrating Peptide Transporter

<400>  46

Arg Arg Arg Arg Arg Arg Arg 
1               5           


<210>  47
<211>  8
<212>  PRT
<213>  Artificial

<220>
<223>  Arginine-Rich Cell-Penetrating Peptide Transporter

<400>  47

Arg Arg Arg Arg Arg Arg Arg Arg 
1               5               


<210>  48
<211>  9
<212>  PRT
<213>  Artificial

<220>
<223>  Arginine-Rich Cell-Penetrating Peptide Transporter

<400>  48

Arg Arg Arg Arg Arg Arg Arg Arg Arg 
1               5                   


<210>  49
<211>  16
<212>  PRT
<213>  Artificial

<220>
<223>  Arginine-Rich Cell-Penetrating Peptide Transporter


<220>
<221>  MOD_RES
<222>  (2)..(2)
<223>  Xaa = Acp

<220>
<221>  MOD_RES
<222>  (4)..(4)
<223>  Xaa = Acp

<220>
<221>  MOD_RES
<222>  (6)..(6)
<223>  Xaa = Acp

<220>
<221>  MOD_RES
<222>  (8)..(8)
<223>  Xaa = Acp

<220>
<221>  MOD_RES
<222>  (10)..(10)
<223>  Xaa = Acp

<220>
<221>  MOD_RES
<222>  (12)..(12)
<223>  Xaa - Acp

<220>
<221>  MOD_RES
<222>  (14)..(14)
<223>  Xaa = Acp

<220>
<221>  MOD_RES
<222>  (16)..(16)
<223>  Xaa = Acp

<400>  49

Arg Xaa Arg Xaa Arg Xaa Arg Xaa Arg Xaa Arg Xaa Arg Xaa Arg Xaa 
1               5                   10                  15      


<210>  50
<211>  12
<212>  PRT
<213>  Artificial

<220>
<223>  Arginine-Rich Cell-Penetrating Pepetide Transporter


<220>
<221>  MOD_RES
<222>  (2)..(2)
<223>  Xaa - 6-aminohexanoic acid

<220>
<221>  MOD_RES
<222>  (5)..(5)
<223>  Xaa = 6-aminohexanoic acid

<220>
<221>  MOD_RES
<222>  (8)..(8)
<223>  Xaa = 6-aminohexanoic acid

<220>
<221>  MOD_RES
<222>  (11)..(11)
<223>  Xaa = 6-aminohexanoic acid

<400>  50

Arg Xaa Arg Arg Xaa Arg Arg Xaa Arg Arg Xaa Arg 
1               5                   10          


<210>  51
<211>  15
<212>  PRT
<213>  Artificial

<220>
<223>  Arginine-Rich Cell-Penetrating Peptide Transporter


<220>
<221>  MOD_RES
<222>  (2)..(2)
<223>  Xaa = 6-aminohexanoic acid

<220>
<221>  MOD_RES
<222>  (5)..(5)
<223>  Xaa = 6-aminohexanoic acid

<220>
<221>  MOD_RES
<222>  (8)..(8)
<223>  Xaa = 6-aminohexanoic acid

<220>
<221>  MOD_RES
<222>  (11)..(11)
<223>  Xaa = 6-aminohexanoic acid

<220>
<221>  MOD_RES
<222>  (14)..(14)
<223>  Xaa = 6-aminohexanoic acid

<400>  51

Arg Xaa Arg Arg Xaa Arg Arg Xaa Arg Arg Xaa Arg Arg Xaa Arg 
1               5                   10                  15  


<210>  52
<211>  12
<212>  PRT
<213>  Artificial

<220>
<223>  Arginine-Rich Cell-Penetrating Peptide Transporter


<220>
<221>  MOD_RES
<222>  (2)..(2)
<223>  Xaa = 6-aminohexanoic acid

<220>
<221>  MOD_RES
<222>  (5)..(5)
<223>  Xaa = beta-alanine

<220>
<221>  MOD_RES
<222>  (8)..(8)
<223>  Xaa = 6-aminohexanoic acid

<220>
<221>  MOD_RES
<222>  (11)..(11)
<223>  Xaa = beta-alanine

<400>  52

Arg Xaa Arg Arg Xaa Arg Arg Xaa Arg Arg Xaa Arg 
1               5                   10          


<210>  53
<211>  15
<212>  PRT
<213>  Artificial

<220>
<223>  Arginine-Rich Cell-Penetrating Transporter

<400>  53

Arg Ala Arg Arg Ala Arg Arg Ala Arg Arg Ala Arg Phe Phe Cys 
1               5                   10                  15  


<210>  54
<211>  15
<212>  PRT
<213>  Artificial

<220>
<223>  Arginine-Rich Cell-Penetrating Peptide Transporter

<400>  54

Arg Gly Arg Arg Gly Arg Arg Gly Arg Arg Gly Arg Phe Phe Cys 
1               5                   10                  15  


