!""# # " " 479. Retroviral V ector-Mediated Expression of Vector-Mediated Therapeutic Levels of Canine Factor IX in Adult and Neonatal Mice Lingfei Xu,1 Mark S. Sands,2 Cuihua Gao,1 Katherine P. Ponder.1 1 Internal Medicine and Biochemistry and Molecular Biophysics, Washington University School of Medicine, St. Louis, MO, United States; 2Internal Medicine and Biochemistry and Genetics, Washington University School of Medicine, St. Louis, MO, United States. C 2 F"
? F -?F/ '
" T&H ?F E
&9H
0 G
# -#D./"
-@./ ?F ' #D."
@. " α8" ? F-?F/$*' 0 "
? B"
2% E
! -C?/ '
,5 % C? &
8," ' ,389: -4I 838989 4%/ @. E
. =9 =B 6: C?
! ?F
>D(' " ?F ?F =8±9= μ% -B9H I *M=/ 6
85±99& μ% -=:H / ; ?F
)
"?F ,9; μ% 8
5&±8: μ% ;
* 3
. E @. C?
@.
-58±=BH/
G * &;2D%B E
@.
8B389;4 -8B38989 4%/ ,"= -*M89/ ! ?F ;6±,8 μ% -86:H / B
56±=B μ% -8::H / B
* 89
?F
! 0 ?F
0
C 2
(
<! * C ? DF
480. Sustained Correction of Hemophilia B Phenotype Following Intravascular Delivery of AA VV ector to AAV Vector Skeletal Muscle Valder R. Arruda,1 Hansell Stedman,2 Nichols C. Timothy,4 Mark Haskins,3 Joerg Schuettrumpf,1 Roland W. Herzog,1 Yi-Lin Liu,1 Kristi Wagner,1 Linda Couto,5 Katherine A. High.1 1 Pediatrics, The Children′s Hospital of Philadelphia, Philadelphia, PA, United States; 2Surgery, University of Pennsylvania School of Medicine, Philadelphia, PA, United States; 3Pathology and Medical Genetics, Univeristy of Pennsylvania School of Veterinary, Philadelphia, PA, United States; 4Pathology and Laboratory Medicine, University of North Carolina, Chapel Hill, NC, United States; 5Avigen, Inc., Alameda, CA, United States. ''.,"
"
?F 2
Molecular Therapy Vol. 5, No. 5, May 2002, Part 2 of 2 Parts Copyright © The American Society of Gene Therapy
?F ''.
8 , ?F % -# .7(,; ,999/ % -2 D47;:8 ,998/ 1 E ' ''."
2 % 1 "
0
2 3
E
-D!/ !
''.
-*#
,76=58555/0 E
,53898,% ''.",
?F *
?F
699 % =9 B99 ;99% -L 89H ?F / D
3 ,6
?F
G
! 85",8 - 78B"8: /
B
- 6=/
- 8,; /
? # E
&9%I 89" ?F
3
D! ?F 3 3
.
?F 6=% -Q 8H / =,
''."?FI 6
E
3
?F #
!@ # E I
!@
?F -8"B 24/
6
D!
"
D!
"
$ C $ 1 '
481. Strain-Specific Differences in the Generation of an Anti-Factor VIII Immune Response in Hemophilic Mice Treated with Adenoviral Gene Therapy Fiona E. M. Grant,1 Brian Brown,1 Chang Xin Shi,2 Frank Graham,2 Shawn Tinlin,1 Alexis McKinven,1 David Lillicrap.1 1 Pathology, Queen′s University, Kingston, ON, Canada; 2Biology, McMaster University, Hamilton, ON, Canada. &;2D%B?.
E ?. C
" .
.
0
C ' .
&;2D%B &;2D% B"2'D2% 2'D2% Δ>8Δ>= 2"
"
?. -2$$ ?./
1 #. "
S157