!" " $ 1 . # "
-*'($'S 7 *./
(
@ '
' 8 855: 4 < ( 0 #
- /$ ′
E . )
@ + #$ " . (
@ '
)
# % @ '"8=8 0 #
4 8=99 + ' * + *+899,8
1215. Retroviral Mediated T ransfer of Ankyrin 2.2 to Transfer nb/nb Spherocytosis Mutants and Engraftment in W/Wv Recipients Results in a New Hemolytic Anemia with Markedly Distorted Red Cell Shape Pamela S. Becker,1 Gerri J. Dooner,1 Helen M. Valinski,1 Berri Jacque,1 Patrick G. Gallagher,3 Bernard G. Forget,3 Jane E. Barker.2 1 Div. of Gene Therapy, Dept. of Medicine, Univ. of Massachusetts Med. School, Worcester, MA, United States; 2The Jackson Laboratory, Bar Harbor, ME, United States; 3Hematology Sect., Dept. of Int. Medicine, Yale Univ. Sch. of Medicine, New Haven, CT, United States.
-C(/ -%/
-,;9 / $*' - 8"6=B&/ =V ,, - 6=&9" &:;9 6B,8"&89:/
8 ,, = 8" " ,, -8"% ',,/
2'D2% 022B?8"% -%/
0
1
- / % 0
"
-* / 2'D2%
-D"/ "
* % D"
022B?8J%J -8899 /
022B?8"0%0 -0% 0 / * 8"% ',,
- / 2'D2% C -* =59 69,/ -* 8,H 8;H/ C * %
022B?8J%J :
8:"==%
&;":&% * 1
E
-
/ # 0%0 %
88
C 8&9 J%" =B -($/ B8; J%" 6BH G
0%0 * % C88;J%",6&;5J%"B9H 3
*
(
G
*
*
($(
3
Molecular Therapy Vol. 5, No. 5, May 2002, Part 2 of 2 Parts Copyright © The American Society of Gene Therapy
68
= 0 G ,,
=
3
%
G 68 =
!" " 1216. Hematopoietic Stem Cells-Directed Gene Therapy: Chronic Granulomatous Disease Maciej Wiznerowicz, Didier Trono. 1 Genetics and Microbiology, University of Geneva, Geneva, Switzerland. C -C(/
" C(
3
3 3 C( "
" " G
" C( # "
D @C
$ 3 " 3
58 3
*'$!C 3 $88 ?! 42 $=6J 58"?! $88"?!
- / -*)$% /
?! 3
3 ?! 3 58 $88
-0!@>/ # 58
$=6J 0!@> 3
*?"
3 C( AD(?-AJ (8J D" ?=D"/
58"?! $88"?!
C(
?! 3
? ?!
$*' 58 3
C(
58 3 "%" -2B"/ @
2B" G
*'$!C 3
-*2 $C@ / - / ) $
1217. Amelioration of Murine β-thalassemia Following Stem Cell Gene T ransfer with a Human γ-Globin Lentiviral Transfer Vector Derek A. Persons,1 Phillip W. Hargrove,1 Esther R. Allay,1 Hideki Hanawa,1 Arthur W. Nienhuis.1 1 Division of Experimental Hematology, St. Jude Children′s Research Hospital, Memphis, TN, United States. )
C? β" -! 2 5;7=,;& ,998/ -($/-2 *#
B78;;,999 ? 2 5;7689 ,998/ γ" γ" ($ " β9" β" 0
γ" 8; 1 6 = ,
β" 1
β" -8;5 / ' γ"
S395
!" "
, =′ -=′ @>/ γ" =′ β"
γ"
.
.(."
*C= =
'
=′ @>
? ?'(
γ" 3 :9H -M6 / "
γ" @*'
5",9H α" @*'
γ"
B " '
3 β"
γ" 3
=′ @> =′ β" γ" 3
?'( 3 #
"
-M:/
β" C 5, ± 9,
γ"
-M5/ γ" -;BH ± 6=/
C C
89: ± 9= 8,& 3
59H# α,% γ,
89"=9H C
3 ,9"=&H C γ" γ" β"
($
1218. Restoration of Correct Splicing by LentivirusDelivered SnRNAs in Cells Expressing β-thalassemic Pre-mRNAs Marla M. Vacek,1 Hong Ma,2 Tal Kafri,2 Ryszard Kole.1,3 1 Curriculum in Genetics and Molecular Biology, University of North Carolina at Chapel Hill, Chapel Hill, NC, United States; 2 Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, United States; 3Lineberger Cancer Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, United States. β" B&6 ;9& ;6& β" &′ =′ ,
β" @*'
" β" 0
@*' 1 =′ β" "@*'
"@*'
β" @*' @ 4B 4; @*' 1 &′
=′ '
1
=′ &′
4B 4; @*'
4;
C D A&B,
3 .(,";9& .(,";6& "@*' 3 .(,"B&6 "
4B
1219. T reatment of Canine Cyclic Neutropenia by Treatment Lentivirus-Mediated G-CSF Delivery William R. Osborne,1 Ofer Yanay,1 Simon C. Barry,1 Louis J. Katen,1 Margaret Brzezinski,1 Lisa Y. Flint,1 Jeff Christensen,2 David C. Dale.2 1 Pediatrics, University of Washington, Seattle, WA, United States; 2 Medicine, University of Washington, Seattle, WA, United States. G
3
@ "(?
'
@@D"!! "#."(?"!@>" (* "(? $*' -!! / C. 8 -!@>/ 2 D
,5= 3 $*' C. % 3 .(." 3 .(."
"(?
,6 3
?!
.
C D 3
"(? & %,6 %89B
" *(?"B9 0
;
02
02
′ -'*/ B
86 '* J%" ($ 659J%"6,9 % '*
&569J%"8&59 % ' "(?
8& %
' '*
-O99,/ 66&9J%",9=9 % 8&=99J%"6,89 % '
"(?
,3895 "(?
E (
'* 6
'* &559J% "8559 % 8;899J%",B:9 %
-O99,/ '* "(?
(
-O99&/
" (? "
"(?
6
"
1220. Phenotypic Correction of Fanconi Anemia Group A Hematopoietic Stem Cells Using Lentiviral V ectors Vectors Kaoru Yamada,1 Ali Ramezani,2 Robert G. Hawley,2 Wolfman Ebell,3 Fre Arwert,4 Christopher E. Walsh.1 1 Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, United States; 2Holland Laboratory, American Red Cross, Rockville, MD, United States; 3Department of Hematology and Bone Marrow Transplantation, Charite Hospital, Berlin, Germany; 4Department of Human Genetics, Free University, Amsterdam, Netherlands. ? ' -?'*/ G
?'* $*' "
S396
Molecular Therapy Vol. 5, No. 5, May 2002, Part 2 of 2 Parts Copyright © The American Society of Gene Therapy