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References
1.
Ayuso
E.
,
Mingozzi
F.
,
Montane
J.
,
Leon
X.
,
Anguela
X.M.
,
Haurigot
V.
,
Edmonson
S.A.
,
Africa
L.
,
Zhou
S.
,
High
K.A.
,
Bosch
F.
,
Wright
J.F.
2010 . High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency . Gene Ther. , 17 :503 –510 .
2.
Flotte
T.R.
,
Berns
K.I.
2005 . Adeno-associated virus: A ubiquitous commensal of mammals . Hum. Gene Ther. , 16 :401 –407 .
3.
Lock
M.
,
Alvira
M.
,
Vandenberghe
L.H.
,
Samanta
A.
,
Toelen
J.
,
Debyser
Z.
,
Wilson
J.M.
2010 . Rapid, simple, and versatile manufacturing of recombinant adeno-associated virus vectors at scale . Hum. Gene Ther. , 21 :1259 –1271 .
4.
Mueller
C.
,
Flotte
T.R.
2008 . Clinical gene therapy using recombinant adeno-associated virus vectors . Gene Ther. , 15 :858 –863 .
5.
Simonelli
F.
,
Maguire
A.M.
,
Testa
F.
,
Pierce
E.A.
,
Mingozzi
F.
,
Bennicelli
J.L.
,
Rossi
S.
,
Marshall
K.
,
Banfi
S.
,
Surace
E.M.
,
Sun
J.
,
Redmond
T.M.
,
Zhu
X.
,
Shindler
K.S.
,
Ying
G.S.
,
Ziviello
C.
,
Acerra
C.
,
Wright
J.F.
,
McDonnell
J.W.
,
High
K.A.
,
Bennett
J.
,
Auricchio
A.
2010 . Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration . Mol. Ther. , 18 :643 –650 .
6.
Summerford
C.
,
Samulski
R.J.
1998 . Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions . J. Virol. , 72 :1438 –1445 .
7.
Vandenberghe
L.H.
,
Wilson
J.M.
,
Gao
G.
2009 . Tailoring the AAV vector capsid for gene therapy . Gene Ther. , 16 :311 –319 .
8.
Vandenberghe
L.H.
,
Xiao
R.
,
Lock
M.
,
Lin
J.
,
Korn
M.
,
Wilson
J.M.
2010 . Efficient serotype-dependent release of functional vector into the culture medium during adeno-associated virus manufacturing . Hum. Gene Ther. , 21 :1251 –1257 .
9.
Zhang
H.
,
Xie
J.
,
Xie
Q.
,
Wilson
J.M.
,
Gao
G.
2009 . Adenovirus-adeno-associated virus hybrid for large-scale recombinant adeno-associated virus production . Hum. Gene Ther. , 20 :922 –929 .