gpp4de4

INTRATHECAL*ADMINISTRATION*OF*AAVrh10*CODING*FOR**
β‐GLUCURONIDASE*CORRECTS*BIOCHEMICAL*AND*HISTOLOGICAL*
HALLMARKS*OF*MUCOPOLYSACCHARIDOSIS*TYPE*VII*MICE**
AND*IMPROVES*BEHAVIOR*AND*SURVIVAL*
Author'/'Autora:'
PhD'Thesis'/'Tesi'Doctoral'
Doctorat'en'Bioquímica,'Biologia'Molecular'i'Biomedicina'
Departament'de'Bioquímica'i'Biologia'Molecular'
Universitat'Autònoma'de'Barcelona'
'
2015*
Gemma*Pagès*i*Pi* Assumpció*Bosch*Merino*
Director'/'Directora:'

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CONCLUSIONS!
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CONCLUSIONS(
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+(#+)%7!#(*%)#+!.#!89:!;*$!#)$!-)$)%!#(*%)#+!.#!+<.#&=!6)%,>!
2. 0012! .+! -)%(! (//.6.(#$! $3&#! 001%345! .#! $%&#+,*6.#?! ;%&.#! &#,! =.'(%! &/$(%!
.#$%&'(#)*+!&,-.#.+$%&$.)#!.#!-.6(>!
3. 0012! %&.+(+! -)%(! "?:! $3&#! 001%345! &/$(%! .#$%&'(#)*+! &,-.#.+$%&$.)#!.#!
.--*#)=)?.6&==7!#&@'(!-.6(A!B3.63!6)%%(=&$(+!$)!&!%.+(!.#!C0;+>!!
4. "#$%&$3(6&=! &,-.#.+$%&$.)#! )/! 001%345! ;7! =*-;&%! <*#6$*%(! =(&,+! $)!
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&+$%)67$(+!&#,!)=.?),(#,%)67$(+>!!
5. "#!DEFGHIJ!-.6(A!.#$%&$3(6&=!&,-.#.+$%&$.)#!)/!001%345!&=+)!$%&#+,*6(+!6(==+!
)/!;%&.#!;=)),!'(++(=+>!!
6. "#$%&$3(6&=! &,-.#.+$%&$.)#! )/! 001%345! &63.('(+! $%&#+,*6$.)#! )/! +)-&$.6!
)%?&#+!&/$(%!,%&.#&?(!)/!001!'(6$)%!$)!$3(!;=)),+$%(&->!!
7. K%&#+,*6$.)#! )/! ;%&.#! &#,! =.'(%! 6&#! ;(! &63.('(,! ;7! =*-;&%! .#$%&$3(6&=!
,(=.'(%7!)/!001%345!B.$3!&!,)+(!L5!$.-(+!=)B(%!$3&#!;7!.#$%&'(#)*+!,(=.'(%7!
)/!0012!B.$3!6)-<&%&;=(!(//.6.(#67>!!
8. K3(! .#$%&$3(6&=! ,(=.'(%7! )/! 001%345! 6),.#?! /)%! βM?=*6*%)#.,&+(! $)! NOP! 1""!
-.6(!=(&,+!$)!&!;($$(%!)*$6)-(!B3(#!-.6(!&%(!.#Q(6$(,!&$!&!7)*#?(%!&?(!&#,!
&/$(%!&!=)#?(%!$%(&$-(#$!,*%&$.)#>!!
9. 0!,)+(!)/!R>FE!S!4544!'?IT?!)/!001%345M:UPG!&,-.#.+$(%(,!.#$%&$3(6&==7!$)!RM
B((TM)=,!NOP!1""!-.6(!6&#!$%&#+,*6(!$3(!DCP!&#,!+)-&$.6!)%?&#+!)/!NOP!1""!
-.6(! &#,! &$$&.#! βM?=*6*%)#.,&+(! &6$.'.$7! =('(=+! $3&$! <%)'.,(! $3(%&<(*$.6!
;(#(/.$>!!

Conclusions)
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$3(! ,(6%(&+(! &#,! ('(#! #)%-&=.V&$.)#!)/! $3(! <&$3)=)?.6&=! ;.)63(-.6&=!
3&==-&%T+!)/!$3(!,.+(&+(!)/!NOP!1""!-.6(!.#!$3(!DCP!&#,!+)-&$.6!)%?&#+>!K3(+(!
.#6=*,(! H0NOM4! &66*-*=&$.)#A! =7+)+)-&=! '(+.6=(! (#=&%?(-(#$A! &+$%)?=.)+.+!
&#,!+(6)#,&%7!(=('&$.)#!)/!=7+)+)-&=!(#V7-&$.6!&6$.'.$7>!
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&6$.'.$7!&#,!$3(!;.)63(-.6&=!6)%%(6$.)#!&$$&.#(,>!
12. K3(! .-<%)'(-(#$!)/! $3(!<37+.6&=A! 6)?#.$.'(! &#,! (-)$.)#&=! 63&%&6$(%.+$.6+! )/!
NOP! 1""! -.6(! <%)'.,(,! ;7! $3(! $%(&$-(#$! &==)B(,! &! ?(#(%&=! .-<%)'(-(#$! .#!
$3(!-)*+(!($3)?%&-!&#,!&!?&.#!)/!/*#6$.)#!.#!$3(.%!&;.=.$7!$)!+B.->!!
13. "#$%&$3(6&=! 001%345M:UPG! $%(&$-(#$! &$$&.#(,! &! 45EW! .#6%(&+(! .#! NOP! 1""!
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