-~
`
` WIPO
`
`WORLD
`INTELLECTUAL PROPERTY
`ORGANIZATION
`
`DOCUMENT MADE AVAILABLE UNDER THE
`PATENT COOPERATION TREATY (PCT)
`PCT/JP2011/070318
`International application number:
`
`International filing date:
`
`31 August 2011 (31.08.2011)
`
`Document type:
`
`Document details:
`
`Certified copy of priority document
`
`Country/Office:
`Number:
`Filing date:
`
`JP
`2010-196032
`01 September 2010 (01.09.2010)
`
`Date of receipt at the International Bureau:
`
`05 October 2011 (05.10.2011)
`
`Remark: Priority document submitted or transmitted to the International Bureau in compliance with Rule
`17.1(a),(b) or (b-bis)
`
`34, chemin des Colombettes
`1 2 I I Geneva 20, Switze1·1and
`
`www.wipo.int
`
`

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`(t~~-'f::s'(Jtlt 1] ~~01ffl0ffl WO 2006/000057
`(t~~-'f::s'(Jtit2] ~~01ffl0ffl WO 2004/048570
`(t~~-'f ::stlt't 3 ] *~1~~-'f01m0ffl US 2 0 10/01 6 8 212
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`P, et al,, Genomics 1988; 2: p, 90-95
`(1M~~-'f::s'(lt'tl] Monaco A,
`(1M~~-'f::s'(Jtit2] Matsuo M,, Brain Dev 1996; 18: p, 167-172
`(1M~~-'f::s'(lt't3] Wilton S, D,, et al,, Molecular Therapy 2007: 15: p, 1288-9
`6
`(1M~~-'f::s'(lt't4] Annemieke Aartsma-Rus et al,,
`12: S 71-S 77
`(1M~~-'f::s'(Jtit5] Linda J, Popplewell et al,,
`vol, 20, no,
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`
`~N/
`I
`O=P-NH2
`I
`~
`
`0
`
`1::: l--1/7- t--071 ✓J!"fii"-O)m-53,m:r.:7'/ ✓ 0)5' *Ji1miJ,l:)m-32,..__,5s=m§L~J;tm-3s,..__,
`[9]
`5s,m§0)3{:7 v:Jt+ FiJ,l:)~QJ!'ic:YUr~:tBflliEtq~ij[£J!'ic:YUiJ,l:)~Q, iwic[ll ,..__, [8l O)P-f:h
`iJ, n:in~ic~O) Y ✓ +t ✓ 7-:Jt u -::1-"'?- 0
`[1 oJ
`J!'ic:YU:m~2,..__,31 iJ, I:)~ QttJ: !J ~tR ~ :tiQ P-f:tiiJ, 1 ~ O)ij[£J!'ic:YU iJ, I:)~ Q, iwic [1
`l ,..__, [8l O)P-f:tiiJ,nJn~ic~O)Y ✓ +t ✓ 7-:Jt u -::1-"'?-◊
`[11J
`J!'ic:YU:m~11- 11, 23, 29Bzr,35iJ,l:)~QttJ:!J~tR~:hQ1t>-f:tiiJ,1~r~~i"ij[£
`J!'ic:YUiJ, I:) ~Q, iwic [1J ,..__, [8l O)P-f:titJ,n1n~ic~O)Y ✓ +t ✓ 7-:Jt u -::1-"'?-◊
`[12J
`J!'ic:YU:m~11JOi3 5 r~~i"ij[£J!'ic:YUiJ, I:)~ Q, iwic [1J ,..__, [8l O)P-f:tiiJ, n:in~ic~O)
`7 ✓ +t ✓ 7-7t9-::l'"'?-◊
`iwic [1J ,..__, [12J O)P-f:tiiJ,nJn~ic~O)Y ✓ +t ✓ 7-:Jt u -::1-"'?-, -i°O)~~Etqr~~-'f~
`[13J
`iiJ~~~ij[)Zti7J<fO!lo/J~1f~11/J>t7t t 9 Q, ffn1/ 7- f-- O 7 1 -mmffl ~~tJl/J>t!lo/Jo
`(¥€.BJ=] 0)~1J:~]
`(0014]
`::zls;:¥£BJ=j 0) 7 ✓ +t ✓ 7-:Jt 9 -::1'"'?- t~ J: !J, 1/ 7- f-- o 7 1 ✓J!"fii"-0):r.:7 '/ ✓ 5 3 0) 7- :\=- '>' I:::'
`✓ '.~l~ rm~11$t~~iJ.Ji" Q 2: t iJ~iiJ~~'"f db Qo ~ f::_,
`::zis;:¥€.BJ=j 0) ~~tJl/J>t!lo/J~N:--1¥-i" Q 2: tr~
`J: !J, -r ::z. 1/ .:r. ✓ 5{2:f'lffn1/ 7- f-- O 7 1 -O)Jitf;:~, ~1J:~E(qt~!lWX9 Q 2: t iJ~-Z: ~ Qo
`( CKIOO 0) M $.~~BJ=]]
`(0015]
`(CK! 1] 1::: I--tfi't¥tffn~J!t*Hll®:t1i: (RD*Hll®) t~:8~t Q 1/7' f-- o 71 ✓J!"fii"-:r.:7 '/ ✓ 53
`0)7-:\=-o/l:::' ✓ ~~-$:~~i"CKl'"fdbQo
`(CK! 2] 1::: I-- IEm'tJl*IEE**lMtlf*Hll® (TI G- l l 9*Hll®) r~ 1::: I-- my oDJ!'lii"- ~J.JJd_,, --C
`ffn~*Hll®t~7}1t~itJ L, f::*Hll®t~:8 ~t Q 1/ 7- f-- o 7 1 ✓J!,fii"-0):r.:7 '/ ✓ 5 3 0) 7- :\=- '>' I:::'
`✓ :7''~11$ ~~i"CKl'"f db Q o
`(CK! 3] 1::: l--DMD,~,::t!tEE**lME#=*Hll® (50 l 7*Hll®) r~ 1::: I-- myoDJ!'lii"-~J.JA L, --Cifn~
`*Hll® r~ 7t1t~itJ L, f:: *Hll® r~ :8 ~ t Q 1/ A I-- 0 7 ,{ ✓Jg "fii"-0) :r. :7 ') ✓ 5 3 0) A :\=- '1/ I:::' ✓ :7''
`~11$ ~~i"CKl'"f db Q o
`(¥€.BJ=]~ ~1ft!I9 Q f:: ~ O)jf;~]
`(0016]
`1. 7 ✓ +t ✓ _:;z_:;t 9 -::1'"'?-
`1::: f-- 1/ 7- f-- o 7 1 ✓J!"fii"-O)m-5 3,m § O):C :7 'J ✓ 0) 7- :\=- '>' I:::' ✓ :7'' ~ iiJ~~t~ 9
`::zis;:¥€.BJ=]ri,
`QY ✓ +t ✓ 7-7t9-::l'"'?-1:db-::>--C, 1::: l--1/7- t--071 ✓J!"fii"-O)m-53,m:r.:7'/ ✓ 0)5'
`*JilmiJ,l:)m-31,..__,53,m§, m-31,..__,54,m§, m-31,..__,55,m§, m-31,..__,5s,m§, m-31,..__,57,m§, m-
`31,..__,58,m§, m-32,..__,53,m§, m-32,..__,54,m§, m-32,..__,55,m§, m-32,..__,5s,m§, m-32,..__,57,m§
`, m-32,..__,58,m§, m-33,..__,53,m§, m-33,..__,54,m§, m-33,..__,55,m§, m-33,..__,5s,m§, m-33,..__,57
`:m§, m-33,..__,58,m§, m-34,..__,53,m§, m-34,..__,54,m§, m-34,..__,55,m§, m-34,..__,5s,m§, m-34
`,..__,57,m§, m-34,..__,58,m§, m-35,..__,53,m§, m-35,..__,54,m§, m-35,..__,55,m§, m-35,..__,5s,m§,
`
`

`

`m-35,..__,5Hl~EL m-35,..__,58:m§L m-36,..__,53,m§L m-36,..__,54,m§L m-36,..__,55,m§L m-36,..__,56,m
`§L m-36,..__,51:m §L~Jim-3 6,..__, 5 8,m § 0) 5{ :7 v 7t+ FiJ, I:)~ Q J!§c!ffU 0) P-f ;tiiJ, 1 --::J rc:tBtlffEtq
`~:®:£J!§c!ffUiJ,l:)~Q, Y ✓ +t ✓ _:;z_:;t9-::1-"'?- c.1,,.1T,
`12js;:¥£BJ:IO):;t9-::1-"'?-J tP7)
`, ~fl1tti"Qo
`(0017]
`[l::: l--1/7- f--07,{ ✓ J!~i"-O)m-53,m§O):C'.7'/ ✓]
`2js;:¥£Bmc:8P .. C
`1J!~i"-J tcti, // / J:..J!~i"-J,,JfHc, cDNA, mRNAFru!IHtBzZ,mRNA t
`~~:hQo Yt~ L, < ti, J!~i"-ti, mRNAFru!IHt, HP't, pre-mRNA--Z:~Qo
`1::: f-- // / J:.. tC:8P .. C 1/ 7- f-- o 7 1 ✓J!~i"-tiJ!~i"-~Xp21, 2 tCf¥ff9 Qo 1/ 7- f-- o 7
`1 ✓J!~i"-ti, 3, 0 Mb p 0)-Jr-{ ;:(~1:f L, --C :8 !J, !E~Jo 0) 1::: 1' J!~i"- t L, --C ti~:::kO)J!~i"-
`1: ~ Qo 1!! L,, 1/ 7- I-- o 7 1 ✓J!~i"-0) :::r- F~:titi:b-fiJ, 14 kb tc~~-9, ~~:::r- F~:ti
`ti79ffl0):C:7'/ ✓ tl,,--C1/7- f--07,{ ✓ J!~i"-pgtcj}~l,,--CPQ (Roberts, RG,, et al,
`, Genomics, 16: 536-538 (1993)) o 1/A f--07,{ ✓ J!~i"-O)fi~!lo/11:~Qpre-mRNAri,
`7- 7' 7 -{ 1/ ✓ :7'-~~~t--C 14 kb O),IJX~mRNA ~1.:/JXi" Q o 1::: 1' O)_ff1_:_2:f!l 1/ 7- 1' o 7 1 ✓J!~i"­
`O)ij[£J!§c!ffU ti0~1: ~ Q (GenBank Accession No, NM_004006) o
`t-- O)Jf1.:W1/ A t-- o 7,,, ✓J!~i"-0):r.:7 Y ✓ 5 3 O)ij[£J!§c!ffU ~J!§c!ffu=m~ uc~i" 0
`1:::
`(0018]
`2js;:¥'5Bj:}0)7t 9 -::1'"'?-ti, :r.:7 '/ ✓ 53 0)7- :\=-'>'I:::' ✓ :7''tc J: !J, DMD2:f!l1/ 7- 1' o 7 1 ✓ Jg~
`i"-tc :::r- F ~ ;hQ :7 ✓ J'\'.71{~' BMD2:f!l1/ A I-- 0 7 ,{ ✓ rcc!U£9 Q 2: t ~ § E(q t l,, l 1t~
`~:hktO)'"Z:~Qo~~--c, 2js;:¥'5~0)7t9~"'?-0):C:7'/ ✓ 7':\=-o/l:::' ✓ ~O)tt~t~Q1/
`7- f-- o 71 ✓J!~i"-0):r.:7 '/ ✓ 53tcti, .ff1.:2:f!ltc:~t--Z:ti~ <, 3{~2:f!lt~~:hQo
`3{~2:fYO)t:::f--1/7-f--o71 ✓ J!~i"-O):r.:7y ✓ 53ri, $!{:tE(qtcti, J,,JTO) (a) )Oi (b
`) rc~c~O)~ 9 3{ :7 v7t+ F'"Z:~Qo
`Ca) J!§c!ffU ,m~ 1 O)ij[£J!§c!ffU t :ifBtlff E(q ~:®:£J!§c!ffU iJ, I:)~ Q ~ 9 3{ :7 v 7t+ F t 7- I-- 9 ✓ 1/ ;r.
`✓ t--~~dtt:T--z:1\-{:1'9 11'-1;:(9Q~93{:7 v7t+ F; Bzt,
`(b) J!§c!ffU=m~ 1 O)ij[£J!§c!ffU rcx-t L, l, 9 O%J,,JJ:.O)[o]~•11: ~1f9 Q:®:£J!§c!ffU iJ, I:)~ Q ~ 9 3{
`:7 v7t+ F
`(0019]
`1 ~ 9 3{ :7 v7t+ FJ t ti, DNA)ZtiRNA ~~~9 Q iJ\ *~ L, < ti, RNA
`2js;:BJ:I*H1~$,
`'""(:~Qo
`17- t-- 9 ✓ 1/;J:. ✓ t--~~dtt:T--z:1\--f :1'9 1f-1;:(9Q~93{:7 v7t+ FJ t
`2js;:BJ:I*H1~$,
`ti, fU x. rt, J!§c!ffU =m~ 1 O)ij[£J!§c!ffU t :ifBtlff E(q ~:®:£J!§c!ffU iJ, I:)~ Q ~ 9 3{ :7 v 7t+ F 0)~1'i~
`)Zti~1'i~~ 7'o- :1' t L, --C,
`:::r o .:::.-;\-{ :1' 9 ~--{--tl-1/ ::1 ✓ $, 7'7- :7 J\-{ :f 9 ~--1
`-e--1/ ::1 ✓ $)Zti-!f--lf ✓ J\-{ :1' 9 ~--1-e--1/ ::1 ✓ $~ t·~ fflPQ-:: t re J: !J 1~ I:) :hQ ~ 9
`3{ :7 v7t+ F ~p ') 0 J\-{ :1' 9 ~--{-e'-1/ 3 ✓ O)jj$ t l,, l ti, fUx.rt, II s amb r O Ok & R
`ussell, Molecular Cloning: A Laboratory Manual Vol,
`3, Cold Spring Harbor, La
`b o r at o r y Pre s s 2 0 0 111 Bz Z, 11 Aus u be 1, Cu r rent Pro t o co 1 s i n Mo 1 e cu 1 a r B i o 1 o g y,
`John W
`iley & Sons 1987-1997 11 ~t•tc~c~~;h--CPQjj$~fljffl9Q2: tiJ~'""(:~Qo
`(0020]
`17- t-- 9 ✓ 1/ ;r. ✓ t-- ~~def: J t ti, {&_:;z_ t-- 9 ✓ 1/ ;r. ✓ t-- ~*ftf:, $ 7- t-- 9
`2js;:BJ:I*H1~$,
`✓ 1/ ;r. ✓ f-- ~*ftf:BzZ,1%7- f-- 9 ✓ 1/ ;r. ✓ f-- ~*{tf:O) P9;h 1: t J: Po 1 {&_:;z_ f-- 9 ✓ 1/ ;r. ✓
`I--~*ftf:J ti, fUx.rt, 5XSSC, 5X-r ✓ J\Jvt--m~, 0, 5%SDS, 50%*JvJ:..7~ F, 32
`°CO)*{tf:'""(:~Qo ~k,
`lql,:7_ f-- 9 ✓ 1/;J:. ✓ I--~*ftf:J ti, fUx.rt, 5XSSC, 5X-r ✓ J\
`Jvt--m~, 0, 5%SDS, 50%*JvJ:..7~ F, 42°C)Zti5XSSC, 1% SDS, 50 mM Tris-HCl (
`pH7. 5), 50%*JvJ:..7~ F, 42°CO)*{tf:1:~Qo
`11%7- I-- 9 ✓ 1/;J:. ✓ t--~*ftf:J ti, iU
`x.rt, 5XSSC, 5X-r ✓ J\Jvt--m~, 0, 5%SDS, 50%*JvJ:..7~ F, 50°C)ZtiO, 2XSSC, 0
`, 1 % SDS, 6 5°C O)*{tf:1: ~ Q o 2: ;h I:) O)*{tf:tC:8P--C, w!&~ J:.~j'Q tit' 1% P[o]~•l1: ~1:f
`i" Q ~ 9 3{ :7 v7t+ FiJ~~:IJ$E(q rc1~ I:) :hQ-:: t iJ~Jt/Jff--Z: ~ Q O k ti L,, J\--f :1' 9 ~--1 -e--
`1/ 3 ✓ 0)7- 1' 9 ✓ 1/ ;r. ✓ 1/-tCJlfli" Q~* t l,, --C tiw!&, 7'o- ·::f~&, 7'o- :lO)ffi:
`~- -17t ✓ 5t&, H~fYL :®:~&~O)~~O)~*iJ~~x. l:);h, ~~1f=t!t--Z:~:ht12:;hl:)O)~*
`
`

`

`~:@i'.§:~:jft9 Q 2: t --Z:[o]:;f-JO) 7- f-- 9 ✓ 1/ .:r. ✓ 1/- ~~fii" Q 2: t iJ~iiJ~~--Z: ~ Q o
`(0021]
`~:8, J\-{-:1'9 ~'-{·lt'-1/::1 ✓ t~$Jl)j0):\=-,1 f--~fflPQ~'ei'ti, fUx.t'!Alkphos Direct
`Labelling and Detection System (GE Healthcare) ~fflPQ2:tiJ~'""(:~Qo 2:0)~'ei'ti
`I-- r~~#O)-:J'o I-- :::r Jvt~ L, tc. iJ~v \, tlil L, tc. -:to - :ft 0)-{ ✓ :\=- : i ~-1/ ::1 ✓ ~~
`:\=- '>'
`,
`Bifr-::>tdJt-
`J( ✓ -::lv ✓ ~55°CO)~dtf:T--Z:0, 1% (w/v) SDS~~D-liX~~J\,177-1:~
`~f~, J\-{-:1'9 ~'-{ ;:(L,fc.~9 7.7 v7t+ F~~tti9Q2: tiJ~'""(:~Qo ~QPti, l!§c!ffU=m
`~l O)ij[£J!§c!ffU t:if'BflliE(q~ij[£J!§c!ffU 0)~1'i~)Oi~1'i~t~£--:5v \--c 7'o- -:1'~1t~i" Q ~r~,
`$Jl)jO)~j~ (fUx.t'!, PCR5'~9 ✓ :7''~,177- (01/:i • ~'-17:7''/7-t±) ~) ~fflP--C
`it-:to--:1'~1/:::f'q::-1/,3/.:::. ✓ (DIG) 7~Jvl,,fc.~'ei"t~ri, DIG~tfflt~te:\=-,1 I-- (01/:i.
`~'-17 :7'' / 7-t±) ~ ffl P --CJ\-{ -:1' 9 ~'-{ ·ll-1/ ::1 ✓ ~~tti 9 Q 2: t iJ~--Z: ~ Q o
`(0022]
`-1:.ic.1,,JJH~1\-1 :f u ~,-1 ;:Z:-iiJ~~~~ 9 ;1. 7 v7t+ Ft L,--Cri, :if'BfoJ•li.~~Y 7 t--
`r1J .:i:. 7
`--z:~ Q BLAsn~ J: !J, 77 ~ Jv t-- O)J'\ 5' )( -!J - ~ fflv \--CgfJiL, tc. t ~ r~,
`l!§c!ffU=m~1 O)~
`9 7. 7 v 7t+ Ft 9 0 %J,,JJ:., 91 %J,,JJ:., 9 2 %J,,JJ:., 9 3 %J,,JJ:., 9 4 %J,,JJ:., 9 5 %J,,JJ:., 9 6 %
`J,,JJ:., 9 7 %J,,JJ:., 9 8 %J,,JJ:., 9 9 %J,,JJ:., 9 9, 1 %J,,JJ:., 9 9, 2 %J,,JJ:., 9 9, 3 %J,,JJ:., 9 9, 4 %1,,_1
`J:., 99, 5%.1,,JJ:., 99, 6%.1,,JJ:., 99, 7%.1,,JJ:., 99, 8%.1,,JJ:., )Oi99, 9%J,,JJ:.O)[o]~•[i.~1f9
`Q ~ 9 7. 7 v7t+ F ~ ~ ~-:f'Q-:: t iJ~--Z: ~ Qo
`(0023]
`~:8, ij[£J!§c!ffU O)[o]~•fi.ri, jJ- 9 ✓ ,&Z,y Jv-r : i - Jvt~ J: Q 7 Jv:::t' 9 ;:(J:..B LAST
`(Bas
`ic Local Alignment Search Tool) (Proc. Natl, Acad, Sci, USA 872264-2268, 1990; P
`roe Natl Acad Sci USA 90: 5873, 1993) ~fflP--C~JE'""(:~Qo BLAST0)7Jv:::t9;(J:..t~
`£--:5Pfc.BLASTN-?BLASTXtlJfr1:hQ70:7''5'J:..iJ~00¥€.~;h--CPQ
`(Altschul SF, et al:
`J Mol Biol 215: 403, 1990) o BLASTN~fflP--Cij[£J!§c!ffU~M:if'Jf9Q~'ei'ri, ;'{7)(-!J
`-ri, fUx.r'!score = 100, wordlength = 12t9Qo BLASTtGapped BLAST7'o:7''5'J:..~
`ffl v \Q~'ei'ri, :;; -:to :7''5' J:.. 0)77 ~ Jv t-- }\ 5' )( - !J - ~ ffl v \Q 0
`(0024]
`:r.7'/ ✓ 530)5' *Ji/miJ\l:)m-31,..__,53,m§L m-31,..__,54,m§L m-31,..__,55,m§L m-31,..__,56,m§
`, m-31,..__,57,m§, m-31,..__,58,m§, m-32,..__,53,m§, m-32,..__,54,m§, m-32,..__,55,m§, m-32,..__,56
`:m§, m-32,..__,57,m§, m-32,..__,58,m§, m-33,..__,53,m§, m-33,..__,54,m§, m-33,..__,55,m§, m-33
`,..__,56,m§, m-33,..__,57,m§, m-33,..__,58,m§, m-34,..__,53,m§, m-34,..__,54,m§, m-34,..__,55,m§,
`m-34,..__,56,m§, m-34,..__,57,m§, m-34,..__,58,m§, m-35,..__,53,m§, m-35,..__,54,m§, m-35,..__,55,m
`§, m-35,..__,56,m§, m-35,..__,57,m§, m-35,..__,58,m§, m-36,..__,53,m§, m-36,..__,54,m§, m-36,..__,
`5 5,m §, m-3 6,..__,5 6,m §, m-36,..__,51:m § ,&r,m-3 6,..__, 5 8,m § 0) ;1. 7 v7t+ FiJ\ I:)~ Q l!§c!ffU r~:if'B
`flliEtl ~ij[£J!§c!ffU 0)1,U ~ J,,_1 T O)f'H~~i" o
`
`

`

`!l
`
`5' TCCGGTTCTGi\i\GGTGTTCTTGTA···J'
`
`5 t
`
`G
`5,
`
`···CTCCGCTTCTGi\AGGTGTTCTTGTA ---:3,
`
`--CCTCCCGTTCTGAi\GGTGTTCTTCTA--:J'
`···GCCTCCGGTTCTGi\AGGTGTTCTTGTA···:3,
`
`5,
`
`··· TGCCTCCGGTTCTGAAGCTGTTCTTGT/\ 3,
`
`5' CTCCGGTTCTGi\AGCrl'GTTCTTGT···J'
`
`f)
`
`CCTCCCGTTCTGJ\i\GGTGTTCTTGT·--3'
`
`G --GCCTCCGLTTCJ'Ci\AGVfLTTCTTCT--:J'
`
`5,
`
`···TGCCTCCGGTTCTGAAGGTGTTCTTGT· :3
`
`5, CCGGTTCTGA/\GGTGTTCTTG ?, '
`
`5' CTCCGCTTCTGi\AGGTCTTCTTG··•:;.
`
`5' CCTCCGGTTCTGA/\GGTGTTCTTGJ'
`
`f)
`
`GCCTCCGCTTCTGi\AGCfCTTCTTG·--3'
`
`s· CCGGTTCTGAi\GGTGTTCTT3'
`
`5' CCTCCCGTTCTGA/\GGTGTTCTT:;.
`
`G
`
`--GCCTCCGLTTCJ'Ci\AGVfLTTCTT -- '.1
`
`f)
`
`····rcc('TCCC(;TTCTGAACG 1·(;TTCl. ! -- .:,
`
`5. CCGGTTCTGA/\GGTGTTCT:1 ·•
`
`5.
`
`···TCCGGTTCTGi\/\GGTGTTCT 3'
`
`5' CCTCCGGTTCTGA/\GGTGTTCT<l'
`
`5' GCCTCCGGTTCTG/\AGCTGTTCT:;.
`
`Fl
`
`5.
`
`··· TCCGGTTCTG/\\GGTGTTC:1 ·•
`
`!l
`f:J
`
`5' GCCTCCGGTTCTGA/\GGTGTTC<l'
`
`5' TGCCTCCGGTTCTGAACGTGTTC:;.
`
`

`

`(0025]
`2js;:¥'5Bj:}0)7t 9 :::f'"7-ri, Yt~ l,, < ri, 1::: f-- 1/7' f-- o 71 ✓ J!"fii"-O)m-53,m:r.:7 '/ ✓ 0)5
`' *Ji/miJ"l:)m-32,..__,5s,m§ (J!'ic!ffU=m~11), m-33,..__,5s,m§ (]!'ic!ffU=m~17), m-34,..__,5s,m§ CJ!'ic!ffU
`=m~23) , m-35,..__,5s,m§ CJ!'ic!ffU=m~29) xrim-3s,..__,5s,m§ CJ!'ic!ffU=m~35) 0)7.:7 v7t+ F
`iJ, I:) -/J:. Q J!'ic!ffU 0)" ,-f :tiiJ, 1 ~ r~:tBflliEtl-/J:.ij[£J!'ic!ffU iJ, I:) -/J:. Q 0
`3lf~L,<ri, 2js;:¥£BJ:I0)7t9:::t"7-ri,
`1::: l--1/7- t--07,,, ✓ J!"fii"-O)m-32,..__,5s,m§ (J!'ic!ffU
`=m~ 11) xrim-3 s,..__, 5 s=m § CJ!'ic!ffU=m~3 5) 0) 7. :7 v7t+ FiJ, I:) -/J:. Q J!'ic!ffU 0) P-f:tiiJ, 1 ~ r~:tB
`flliE(q-/J:.ij[£J!'ic!ffU iJ, I:) -/J:. Q t 0) -z: dfJ Q o
`(0026]
`l-- 1/ 7- t-- o 7,., ✓J!"fii"-O)m-5 3,m § O):r. :7 'I ✓ 0) 7- =f- ,1 I:::' ✓ :7''~ iiJ~~r~ 9 Q J t ri
`r 1:::
`1::: l--1/A f--07,{ ✓J!"fii"-O)!i~W (fUx.rt, pre-mRNA) O):C:7'/ ✓ 53r~:tB~9Q"fi~tr
`'
`r~2js;:¥£BJ:I0)7t 9 :::t"7-iJ~*a'Ei'9 Q-:: tr~ J: !J, ~t!i~WiJ~_:;z_ 77 ,,r 1/ ✓ :7'-~~~ttc.~r~,
`fU;t rt:r. :7 'I ✓ 5 2iJ~)::~ L, fc.DMD,~,~O)~'Ef, :r. :7 'I ✓ 510) 3' *Ji/mr~:tB ~9 Qij[£J!'ic!ffU 0)
`,mur~ :r. :7 Y ✓ 5 4 0) 5, *Ji/mr~:tB ~ 9 Q ij[£J!'ic!ffU iJ~JI,ta L, --c :8 !J, ::1 F ✓ 0) 7 v-J.>. 1/ 7
`3,
`1' iJ~~2:-::> --CP-/J:.P,ax;~mRNAiJ~lf;,ax;~ tl,Q 2: t ~~~9 Qo
`VE:-:> --C, 2js;:¥£BJ:} 0)7t 9 :::f'"7- ri, 1::: 1' 1/ 7- f-- o 7 1 ✓ J!"fii"-0):r. :7 'J ✓ 5 3 0) 7- =f- ''/ I:::' ✓
`:7''~ iiJ~~r~9 Q ~ft !J' ly-J/ ''/ I-- J!'ic!ffU r~xtL, --Cl OO%:tBflliE(q-/;J:.ij[£]!'ic!ffU ~1f L, --C lt ,-/J:. < l
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