"8 "!" ! )
89 μ -8389; / " C(. C#.
- (C!>/ > β" - (CN/ !
-('!(I 6 9 M /
E
('!(8;±9,,
,B±9,
E #
(C!>
('!(9,±98,
-O99998(CN/ 89±9=
-O9999& (CN/ E @
E
E
3
(C!> (CN
-,9 ± 9= (C!> ,6 ± 9, (CNI = /
(C!>
E 3 -8 μ/
,, ± 9, " 3
1339. Ribozyme-Mediated Striatal Reduction of Huntingtin in Adult Mice Edgardo Rodriguez,1 Lorraine Hamilton,2 Eileen Denovan-Wright,2 Alfred S. Lewin,3 Ronald J. Mandel.1 1 Neuroscience, University of Florida College of Medicine, Gainesville, FL, United States; 2Pharmacology, Dalhousie University, Halifax, Nova Scotia, Canada; 3Molecular Genetics, University of Florida College of Medicine, Gainesville, FL, United States. C -C$/
G
-'/ 3 &′" C$ C$ =& "
" -S/ 3 *" - / S 3 -*/
C$ C 1 3
@
′
0
"
, - ''.,/ @*'" G - G /
C$ ( G
"
@*'
A A C$B C$; G C$ 3 8 1
C$8 G 3 8 1 G 1
3
@*' 3
3 8 C$ @*' (
G ?
C$; G ''., @>("?!
4 ? . 0 E
, 3 , 8988 4% C$;" ''., -B":
/ @B% , 3 ?! E
" E ' ="&
′
?!"
''." G " E
@*'
@*'
1 @ "!@ ) B9 H
@*' G
0 C$; G
@*'
@*' " C$
Molecular Therapy Vol. 5, No. 5, May 2002, Part 2 of 2 Parts Copyright © The American Society of Gene Therapy
1340. Lentiviral V ectors for In V ivo Gene Therapy of Vectors Vivo Metachromatic Leukokystrophy: Study on the Mechanisms Underlying the Therapeutic Effect Antonella Consiglio,1 Diego Dolcetta,1 Tonia Luca,1 Alessandra Biffi,1 Angela Gritti,2 Antonia Follenzi,3 Angelo Vescovi,2 M. Grazia Roncarolo,1 Luigi Naldini,3 Claudio Bordignon.1 1 San Raffaele-Telethon Institute for Gene Therapy, (H.S.R.TIGET), Scientific Institute H. San Raffaele, Milan, Italy; 2Stem Cell Research Institute, Scientific Institute H. San Raffaele, Milan, Italy; 3Institute for Cancer Research and Treatment, University of Torino, Italy. # D -#D$/
G ' -'@('/ G
0
E '@('"
'
3
G #D$ G 1
E
3
-/
# G
"
E
E 3 ?! '(,"%"
?! E ?! E
E E
G (
G #D$ ' % E
"
G
G ?! E
'
E
3
3 ?!
3 " ?!"
E
#D$
#
*(
S439