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Lili Wang
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Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
GP Gao, MR Alvira, L Wang, R Calcedo, J Johnston, JM Wilson
Proceedings of the National Academy of Sciences 99 (18), 11854-11859, 2002
20052002
Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a …
WW Hauswirth, TS Aleman, S Kaushal, AV Cideciyan, SB Schwartz, ...
Human gene therapy 19 (10), 979-990, 2008
12002008
A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice
Y Yang, L Wang, P Bell, D McMenamin, Z He, J White, H Yu, C Xu, ...
Nature biotechnology 34 (3), 334-338, 2016
6432016
Chronic suppression of heart-failure progression by a pseudophosphorylated mutant of phospholamban via in vivo cardiac rAAV gene delivery
M Hoshijima, Y Ikeda, Y Iwanaga, S Minamisawa, M Date, Y Gu, ...
Nature medicine 8 (8), 864-871, 2002
4692002
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
ML Brantly, JD Chulay, L Wang, C Mueller, M Humphries, LT Spencer, ...
Proceedings of the National Academy of Sciences 106 (38), 16363-16368, 2009
3872009
Adeno-associated virus antibody profiles in newborns, children, and adolescents
R Calcedo, H Morizono, L Wang, R McCarter, J He, D Jones, ML Batshaw, ...
Clinical and Vaccine Immunology 18 (9), 1586-1588, 2011
3462011
Sustained correction of bleeding disorder in hemophilia B mice by gene therapy
L Wang, K Takabe, SM Bidlingmaier, CR Ill, IM Verma
Proceedings of the National Academy of Sciences 96 (7), 3906-3910, 1999
2951999
Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype
R Sarkar, R Tetreault, G Gao, L Wang, P Bell, R Chandler, JM Wilson, ...
Blood 103 (4), 1253-1260, 2004
2712004
Hybrid vectors based on adeno-associated virus serotypes 2 and 5 for muscle-directed gene transfer
M Hildinger, A Auricchio, G Gao, L Wang, N Chirmule, JM Wilson
Journal of virology 75 (13), 6199-6203, 2001
2702001
Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey
LH Vandenberghe, P Bell, AM Maguire, CN Cearley, R Xiao, R Calcedo, ...
Science translational medicine 3 (88), 88ra54-88ra54, 2011
2622011
Efficacy and safety of adeno‐associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy
T Vandendriessche, L Thorrez, A Acosta‐Sanchez, I Petrus, L Wang, L Ma, ...
Journal of Thrombosis and Haemostasis 5 (1), 16-24, 2007
2522007
Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid
LH Vandenberghe, L Wang, S Somanathan, Y Zhi, J Figueredo, ...
Nature medicine 12 (8), 967-971, 2006
2512006
Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver
L Wang, TC Nichols, MS Read, DA Bellinger, IM Verma
Molecular therapy 1 (2), 154-158, 2000
2302000
Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy
L Wang, R Calcedo, TC Nichols, DA Bellinger, A Dillow, IM Verma, ...
Blood 105 (8), 3079-3086, 2005
2142005
Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates
G Gao, Y Lu, R Calcedo, RL Grant, P Bell, L Wang, J Figueredo, M Lock, ...
Molecular Therapy 13 (1), 77-87, 2006
2122006
Impact of pre-existing immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors
L Wang, R Calcedo, P Bell, J Lin, RL Grant, DL Siegel, JM Wilson
Human gene therapy 22 (11), 1389-1401, 2011
2032011
A factor IX-deficient mouse model for hemophilia B gene therapy
L Wang, M Zoppè, TM Hackeng, JH Griffin, KF Lee, IM Verma
Proceedings of the National Academy of Sciences 94 (21), 11563-11566, 1997
2021997
In vivo somatic cell gene transfer of an engineered Noggin mutein prevents BMP4-induced heterotopic ossification
DL Glaser, AN Economides, L Wang, X Liu, RD Kimble, JP Fandl, ...
JBJS 85 (12), 2332-2342, 2003
1802003
Scalable mRNA and siRNA lipid nanoparticle production using a parallelized microfluidic device
SJ Shepherd, CC Warzecha, S Yadavali, R El-Mayta, MG Alameh, ...
Nano Letters 21 (13), 5671-5680, 2021
1772021
Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins
A Auricchio, E O’Connor, D Weiner, GP Gao, M Hildinger, L Wang, ...
The Journal of clinical investigation 110 (4), 499-504, 2002
1752002
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