|
🐜
|
Safety and efficacy of gene transfer for Leber's congenital amaurosis.
32 auth.
A. Maguire,
F. Simonelli,
E. Pierce,
E. Pugh,
F. Mingozzi,
J. Bennicelli,
S. Banfi,
Kathleen A. Marshall,
F. Testa,
E. Surace,
S. Rossi,
A. Lyubarsky,
V. Arruda,
B. Konkle,
E. Stone,
...
Junwei Sun,
J. Jacobs,
L. Dell’Osso,
R. Hertle,
Jian-xing Ma,
T Michael Redmond,
Xiaosong Zhu,
B. Hauck,
O. Zelenaia,
K. Shindler,
M. Maguire,
J. F. Wright,
N. Volpe,
J. McDonnell,
A. Auricchio,
K. High,
J. Bennett
|
11 |
2008 |
11
🐜
|
|
🐜
|
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
32 auth.
C. Manno,
V. Arruda,
G. Pierce,
B. Glader,
M. Ragni,
J. Rasko,
M. Ozelo,
K. Hoots,
P. Blatt,
B. Konkle,
M. Dake,
R. Kaye,
M. Razavi,
A. Zajko,
J. Zehnder,
...
H. Nakai,
A. Chew,
D. Leonard,
D. Leonard,
J. F. Wright,
R. Lessard,
J. Sommer,
M. Tigges,
D. Sabatino,
A. Luk,
Haiyan Jiang,
F. Mingozzi,
L. Couto,
H. Ertl,
K. High,
K. High,
M. Kay
|
10 |
2006 |
10
🐜
|
|
🐜
|
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.
32 auth.
A. Nathwani,
E. Tuddenham,
S. Rangarajan,
Cecilia Rosales,
J. McIntosh,
D. Linch,
P. Chowdary,
A. Riddell,
Arnulfo Pie,
C. Harrington,
J. O’Beirne,
Keith A. Smith,
J. Pasi,
B. Glader,
P. Rustagi,
...
C. Ng,
M. Kay,
Junfang Zhou,
Yunyu Spence,
C. Morton,
J. Allay,
J. Coleman,
S. Sleep,
J. Cunningham,
D. Srivastava,
E. Basner-Tschakarjan,
F. Mingozzi,
K. High,
J. Gray,
U. Reiss,
A. Nienhuis,
A. Davidoff
|
10 |
2011 |
10
🐜
|
|
🐜
|
Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
30 auth.
A. Nathwani,
U. Reiss,
E. Tuddenham,
Cecilia Rosales,
P. Chowdary,
J. McIntosh,
M. della Peruta,
Elsa Lhériteau,
Nishali Patel,
D. Raj,
A. Riddell,
J. Pie,
S. Rangarajan,
D. Bevan,
M. Recht,
...
Yu-min P Shen,
K. Halka,
E. Basner-Tschakarjan,
F. Mingozzi,
K. High,
J. Allay,
M. Kay,
C. Ng,
Junfang Zhou,
M. Cancio,
C. Morton,
J. Gray,
D. Srivastava,
A. Nienhuis,
A. Davidoff
|
10 |
2014 |
10
🐜
|
|
🦁
|
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
F. Mingozzi,
K. High
|
9 |
2011 |
9
🦁
|
|
🐜
|
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
41 auth.
A. Maguire,
A. Maguire,
K. High,
K. High,
A. Auricchio,
J. F. Wright,
J. F. Wright,
E. Pierce,
E. Pierce,
F. Testa,
F. Mingozzi,
J. Bennicelli,
G. Ying,
S. Rossi,
A. Fulton,
...
Kathleen A. Marshall,
S. Banfi,
D. Chung,
J. I. Morgan,
B. Hauck,
O. Zelenaia,
Xiaosong Zhu,
L. Raffini,
F. Coppieters,
E. Baere,
K. Shindler,
N. Volpe,
E. Surace,
Carmela Acerra,
A. Lyubarsky,
T. Redmond,
T Michael Redmond,
E. Stone,
E. Stone,
Junwei Sun,
Jenni Fer Uvellman Mcdonnell,
B. Leroy,
B. Leroy,
F. Simonelli,
J. Bennett,
J. Bennett
|
9 |
2009 |
9
🐜
|
|
🦁
|
Immune responses to AAV vectors: overcoming barriers to successful gene therapy.
F. Mingozzi,
K. High
|
9 |
2013 |
9
🦁
|
|
🦁
|
CD8+ T-cell responses to adeno-associated virus capsid in humans
13 auth.
F. Mingozzi,
M. Maus,
Daniel J. Hui,
D. Sabatino,
S. L. Murphy,
J. Rasko,
M. Ragni,
C. Manno,
J. Sommer,
Haiyan Jiang,
...
G. Pierce,
H. Ertl,
K. High
|
9 |
2007 |
9
🦁
|
|
🐢
|
Emerging Issues in AAV-Mediated In Vivo Gene Therapy
P. Colella,
G. Ronzitti,
F. Mingozzi
|
9 |
2017 |
9
🐢
|
|
🐜
|
Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration
22 auth.
F. Simonelli,
A. Maguire,
F. Testa,
E. Pierce,
F. Mingozzi,
J. Bennicelli,
S. Rossi,
Kathleen A. Marshall,
S. Banfi,
E. Surace,
...
Junwei Sun,
T Michael Redmond,
Xiaosong Zhu,
K. Shindler,
G. Ying,
C. Ziviello,
Carmela Acerra,
J. F. Wright,
J. McDonnell,
K. High,
J. Bennett,
A. Auricchio
|
9 |
2009 |
9
🐜
|
|
🦁
|
Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer.
9 auth.
F. Mingozzi,
Yi-lin Liu,
E. Dobrzynski,
A. Kaufhold,
Jian Hua Liu,
Yuqin Wang,
...
V. Arruda,
K. High,
R. Herzog
|
8 |
2003 |
8
🦁
|
|
🐢
|
AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer
Helena Costa Verdera,
K. Kuranda,
F. Mingozzi
|
8 |
2020 |
8
🐢
|
|
🐜
|
AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness
20 auth.
J. Bennett,
M. Ashtari,
J. Wellman,
Kathleen A. Marshall,
L. Cyckowski,
D. Chung,
Sarah Mccague,
E. Pierce,
Yifeng Chen,
J. Bennicelli,
...
Xiaosong Zhu,
G. Ying,
Junwei Sun,
J. F. Wright,
A. Auricchio,
F. Simonelli,
K. Shindler,
F. Mingozzi,
K. High,
A. Maguire
|
8 |
2012 |
8
🐜
|
