ttr-rb15

1
5’ 3’ TTR-RB15 TTR-RB15 monomer, 2490 nt TTR-RB15 5’ 3’ Self-complementary TTR-RB15 dimer, 4856 nt (A) pAAV2-TTR-RB15 (used as marker) scAAV2-TTR-RB15 Dimer, 4856 nt Monomer, 2490 nt Parent, 3967 nt Not-digested (B) days 1 1 1 1 3 3 3 3 HepG2 Parent scRB15 scRB15- mutant RT-PCR PCR 1 1 1 1 3 3 3 3 HepG2 Parent scRB15 scRB15- mutant RB15 (C) (D) ApoB mRNA in HepG2 (% of inactive RB15-mutant) A d - R B 1 5 - m u t a n t A d - R B 1 5 P a r e n t - A A V 2 - T T R - R B 1 5 - m u t a n t s c A A V 2 - T T R - R B 1 5 s c A A V 2 - T T R - R B 1 5 - m u t a n t 0 25 50 75 100 125 P a r e n t - A A V 2 - T T R - R B 1 5 P=0.005 P=0.001 ns

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(A). (B). pAAV2-TTR-RB15 (used as marker). scAAV2-TTR-RB15. 3’. 5’. TTR-RB15. TTR-RB15 monomer, 2490 nt. Not-digested. Dimer, 4856 nt. Parent, 3967 nt. Monomer, 2490 nt. 3’. TTR-RB15. 5’. Self-complementary TTR-RB15 dimer, 4856 nt. RT-PCR. (C). PCR. scRB15- mutant. scRB15- - PowerPoint PPT Presentation

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Page 1: TTR-RB15

5’3’TTR-RB15

TTR-RB15 monomer, 2490 nt

TTR-RB155’3’

Self-complementaryTTR-RB15 dimer, 4856 nt

(A) pAAV2-TTR-RB15(used as marker) scAAV2-TTR-RB15

Dimer, 4856 nt

Monomer, 2490 ntParent, 3967 nt

Not-digested

(B)

days 1 1 1 13 3 3 3

HepG2

Parent sc

RB15scR

B15-

mutant

RT-PCR PCR

1 1 1 13 3 3 3

HepG2

Parent sc

RB15scR

B15-

mutant

RB15

(C)

(D)

Apo

B m

RN

A in

Hep

G2

(% o

f in

acti

ve R

B15

-mut

ant)

Ad- RB1 5

-mutant

Ad-RB15

Parent

- AAV2-TTR

-RB1 5-mut ant

scAAV2-TTR

-RB15

scAAV2-TTR

-RB15-mutant

0

25

50

75

100

125

Parent-AAV2

-TTR-RB15

P=0.005 P=0.001ns