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The human visual cortex responds to gene therapy-mediated recovery of retinal function.
Published in:
2011
By:
- Ashtari, Manzar;
- Cyckowski, Laura L.;
- Monroe, Justin F.;
- Marshall, Kathleen A.;
- Chung, Daniel C.;
- Auricchio, Alberto;
- Simonelli, Francesca;
- Leroy, Bart P.;
- Maguire, Albert M.;
- Shindler, Kenneth S.;
- Bennett, Jean
Publication type:
journal article
Management of Presumed Endogenous Fungal Endophthalmitis in a Child With Acute Lymphoblastic Leukemia.
Published in:
2017
By:
- Uyhazi, Katherine E.;
- Kolomeyer, Anton M.;
- Gray, Iga N.;
- Traband, Anastasia;
- Kohli, Anita A.;
- O'Brien, Joan M.;
- Maguire, Albert M.
Publication type:
Case Study
Gene therapy restores vision in a canine model of childhood blindness.
Published in:
Nature Genetics, 2001, v. 28, n. 1, p. 92, doi. 10.1038/88327
By:
- Acland, Gregory M.;
- Aguirre, Gustavo D.;
- Ray, Jharna;
- Zhang, Qi;
- Aleman, Tomas S.;
- Cideciyan, Artur V.;
- Pearce-Kelling, Susan E.;
- Anand, Vibha;
- Zeng, Yong;
- Maguire, Albert M.;
- Jacobson, Samuel G.;
- Hauswirth, William W.;
- Bennett, Jean
Publication type:
Article
Subretinal Injection of Voretigene Neparvovec-rzyl in a Patient With RPE65-Associated Leber's Congenital Amaurosis.
Published in:
2019
By:
- Hussain, Rehan M.;
- Tran, Kimberly D.;
- Maguire, Albert M.;
- Berrocal, Audina M.
Publication type:
journal article
Correction: AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models.
Published in:
PLoS ONE, 2015, v. 10, n. 6, p. 1, doi. 10.1371/journal.pone.0129982
By:
- Vasireddy, Vidyullatha;
- Mills, Jason A.;
- Gaddameedi, Rajashekhar;
- Basner-Tschakarjan, Etiena;
- Kohnke, Monika;
- Black, Aaron D.;
- Alexandrov, Krill;
- Zhou, Shangzhen;
- Maguire, Albert M.;
- Chung, Daniel C.;
- Mac, Helen;
- Sullivan, Lisa;
- Gadue, Paul;
- Bennicelli, Jeannette L.;
- French, Deborah L.;
- Bennett, Jean
Publication type:
Article
AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models
Published in:
PLoS ONE, 2013, v. 8, n. 5, p. 1, doi. 10.1371/journal.pone.0061396
By:
- Vasireddy, Vidyullatha;
- Mills, Jason A.;
- Gaddameedi, Rajashekhar;
- Basner-Tschakarjan, Etiena;
- Kohnke, Monika;
- Black, Aaron D.;
- Alexandrov, Krill;
- Zhou, Shangzhen;
- Maguire, Albert M.;
- Chung, Daniel C.;
- Mac, Helen;
- Sullivan, Lisa;
- Gadue, Paul;
- Bennicelli, Jeannette L.;
- French, Deborah L.;
- Bennett, Jean
Publication type:
Article
AAV9 Targets Cone Photoreceptors in the Nonhuman Primate Retina.
Published in:
PLoS ONE, 2013, v. 8, n. 1, p. 1, doi. 10.1371/journal.pone.0053463
By:
- Vandenberghe, Luk H.;
- Bell, Peter;
- Maguire, Albert M.;
- Ru Xiao;
- Hopkins, Tim B.;
- Grant, Rebecca;
- Bennett, Jean;
- Wilson, James M.
Publication type:
Article
Compensatory Cross-Modal Plasticity Persists After Sight Restoration.
Published in:
Frontiers in Neuroscience, 2020, v. 14, p. 1, doi. 10.3389/fnins.2020.00291
By:
- Mowad, Theresa G.;
- Willett, Aimee E.;
- Mahmoudian, Mani;
- Lipin, Mikhail;
- Heinecke, Armin;
- Maguire, Albert M.;
- Bennett, Jean;
- Ashtari, Manzar
Publication type:
Article
Short-term Assessment of Subfoveal Injection of Adeno-Associated Virus-Mediated hCHM Gene Augmentation in Choroideremia Using Adaptive Optics Ophthalmoscopy.
Published in:
2022
By:
- Morgan, Jessica I. W.;
- Jiang, Yu You;
- Vergilio, Grace K.;
- Serrano, Leona W.;
- Pearson, Denise J.;
- Bennett, Jean;
- Maguire, Albert M.;
- Aleman, Tomas S.
Publication type:
journal article
Utility of In Vitro Mutagenesis of RPE65 Protein for Verification of Mutational Pathogenicity Before Gene Therapy.
Published in:
2019
By:
- Yang, Unikora;
- Gentleman, Susan;
- Gai, Xiaowu;
- Gorin, Michael B.;
- Borchert, Mark S.;
- Lee, Thomas C.;
- Villanueva, Adda;
- Koenekoop, Robert;
- Maguire, Albert M.;
- Bennett, Jean;
- Redmond, T. Michael;
- Nagiel, Aaron
Publication type:
journal article
Atypical Retinitis in the Setting of Prior Cytomegalovirus Retinitis.
Published in:
2016
By:
- Sandhu, Harpal Singh;
- Maguire, Albert M.
Publication type:
journal article
Atypical Presentation of Bone Marrow Transplant Retinopathy.
Published in:
Journal of Pediatric Ophthalmology & Strabismus, 2003, v. 40, n. 6, p. 361
By:
- Demirci, Hakan;
- Shields, Jerry A.;
- Shields, Carol L.;
- Maguire, Albert M.
Publication type:
Article
Plasticity of the human visual system after retinal gene therapy in patients with Leber’s congenital amaurosis.
Published in:
Science Translational Medicine, 2015, v. 7, n. 296, p. 1, doi. 10.1126/scitranslmed.aaa8791
By:
- Ashtari, Manzar;
- Hui Zhang;
- Cook, Philip A.;
- Cyckowski, Laura L.;
- Shindler, Kenneth S.;
- Marshall, Kathleen A.;
- Aravand, Puya;
- Vossough, Arastoo;
- Gee, James C.;
- Maguire, Albert M.;
- Baker, Chris I.;
- Bennett, Jean
Publication type:
Article
AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness.
Published in:
Science Translational Medicine, 2012, v. 4, n. 120, p. 1, doi. 10.1126/scitranslmed.3002865
By:
- Bennett, Jean;
- Ashtari, Manzar;
- Wellman, Jennifer;
- Marshall, Kathleen A.;
- Cyckowski, Laura L.;
- Chung, Daniel C.;
- McCague, Sarah;
- Pierce, Eric A.;
- Yifeng Chen;
- Bennicelli, Jeannette L.;
- Xiaosong Zhu;
- Gui-shuang Ying;
- Junwei Sun;
- Fraser Wright, J.;
- Auricchio, Alberto;
- Simonelli, Francesca;
- Shindler, Kenneth S.;
- Mingozzi, Federico;
- High, Katherine A.;
- Maguire, Albert M.
Publication type:
Article
Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey.
Published in:
Science Translational Medicine, 2011, v. 3, n. 88, p. 1, doi. 10.1126/scitranslmed.3002103
By:
- Vandenberghe, Luk H.;
- Bell, Peter;
- Maguire, Albert M.;
- Cearley, Cassia N.;
- Xiao, Ru;
- Calcedo, Roberto;
- Wang, Lili;
- Castle, Michael J.;
- Maguire, Alexandra C.;
- Grant, Rebecca;
- Wolfe, John H.;
- Wilson, James M.;
- Bennett, Jean
Publication type:
Article
Safety and Efficacy of Subretinal Readministration of a Viral Vector in Large Animals to Treat Congenital Blindness.
Published in:
Science Translational Medicine, 2010, v. 2, n. 21, p. 1, doi. 10.1126/scitranslmed.3000659
By:
- Amado, Defne;
- Mingozzi, Federico;
- Hui, Daniel;
- Bennicelli, Jeannette L.;
- Zhangyong Wei;
- Yifeng Chen;
- Bote, Erin;
- Grant, Rebecca L.;
- Golden, Jeffrey A.;
- Narfstrom, Kristina;
- Syed, Nasreen A.;
- Orlin, Stephen E.;
- High, Katherine A.;
- Maguire, Albert M.;
- Bennett, Jean
Publication type:
Article
Nonhuman primate models for diabetic ocular neovascularization using AAV2-mediated overexpression of vascular endothelial growth factor.
Published in:
2005
By:
- Lebherz, Corinna;
- Maguire, Albert M.;
- Auricchio, Alberto;
- Tang, Waixing;
- Aleman, Tomas S.;
- Wei, Zhangyong;
- Grant, Rebecca;
- Cideciyan, Artur V.;
- Jacobson, Samuel G.;
- Wilson, James M.;
- Bennett, Jean
Publication type:
journal article
Adeno-associated virus 8-mediated gene therapy for choroideremia: preclinical studies in in vitro and in vivo models.
Published in:
Journal of Gene Medicine, 2014, v. 16, n. 5/6, p. 122, doi. 10.1002/jgm.2768
By:
- Black, Aaron;
- Vasireddy, Vidyullatha;
- Chung, Daniel C.;
- Maguire, Albert M.;
- Gaddameedi, Rajashekhar;
- Tolmachova, Tania;
- Seabra, Miguel;
- Bennett, Jean
Publication type:
Article
Phenotypic rescue after adeno-associated virus-mediated delivery of 4-sulfatase to the retinal pigment epithelium of feline mucopolysaccharidosis VI.
Published in:
Journal of Gene Medicine, 2002, v. 4, n. 6, p. 613, doi. 10.1002/jgm.302
By:
- Ho, Thucanh T.;
- Maguire, Albert M.;
- Aguirre, Gustavo D.;
- Surace, Enrico M.;
- Anand, Vibha;
- Zeng, Yong;
- Salvetti, Anna;
- Hopwood, John J.;
- Haskins, Mark E.;
- Bennett, Jean
Publication type:
Article
Strain-dependent anterior segment neovascularization following intravitreal gene transfer of basic fibroblast growth factor (bFGF).
Published in:
Journal of Gene Medicine, 2001, v. 3, n. 3, p. 252, doi. 10.1002/1521-2254(200105/06)3:3<252::AID-JGM185>3.0.CO;2-S
By:
- Gupta, Abha R.;
- Dejneka, Nadine S.;
- D'Amato, Robert J.;
- Yang, Zaixin;
- Syed, Nasreen;
- Maguire, Albert M.;
- Bennett, Jean
Publication type:
Article
Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration.
Published in:
Molecular Therapy, 2010, v. 18, n. 3, p. 643, doi. 10.1038/mt.2009.277
By:
- Simonelli, Francesca;
- Maguire, Albert M.;
- Testa, Francesco;
- Pierce, Eric A.;
- Mingozzi, Federico;
- Bennicelli, Jeannette L.;
- Rossi, Settimio;
- Marshall, Kathleen;
- Banfi, Sandro;
- Surace, Enrico M.;
- Sun, Junwei;
- Redmond, T. Michael;
- Xiaosong Zhu;
- Shindler, Kenneth S.;
- Gui-Shuang Ying;
- Ziviello, Carmela;
- Acerra, Carmela;
- Wright, J. Fraser;
- McDonnell, Jennifer Wellman;
- High, Katherine A.
Publication type:
Article
Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer.
Published in:
Molecular Therapy, 2008, v. 16, n. 3, p. 458, doi. 10.1038/sj.mt.6300389
By:
- Bennicelli, Jeannette;
- Wright, John Fraser;
- Komaromy, Andras;
- Jacobs, Jonathan B.;
- Hauck, Bernd;
- Zelenaia, Olga;
- Mingozzi, Federico;
- Hui, Daniel;
- Chung, Daniel;
- Rex, Tonia S.;
- Zhangyong Wei;
- Guang Qu;
- Shangzhen Zhou;
- Zeiss, Caroline;
- Arruda, Valder R.;
- Acland, Gregory M.;
- Dell'Osso, Lou F.;
- High, Katherine A.;
- Maguire, Albert M.;
- Bennett, Jean
Publication type:
Article
881. Safety, Efficacy and Biodistribution of Recombinant AAV2-RPE65 Vector Delivered by Ocular Subretinal Injection.
Published in:
Molecular Therapy, 2006, v. 13, p. S339, doi. 10.1016/j.ymthe.2006.08.970
By:
- Jacobson, Samuel G.;
- Acland, Gregory M.;
- Aguirre, Gustavo D.;
- Aleman, Tomas S.;
- Boye, Sanford L.;
- Schwartz, Sharon B.;
- Cideciyan, Artur V.;
- Zeiss, Caroline J.;
- Komaromy, Andras M.;
- Roman, Alejandro J.;
- Windsor, Elizabeth A. M.;
- Sumaroka, Alexander;
- Pearce-Kelling, Susan E.;
- Conlon, Thomas J.;
- Quihong Li;
- Chiodo, Vincent A.;
- Flotte, Terence R.;
- Maguire, Albert M.;
- Bennett, Jean;
- Hauswirth, William W.
Publication type:
Article
Safety of Recombinant Adeno-Associated Virus Type 2–RPE65 Vector Delivered by Ocular Subretinal Injection.
Published in:
Molecular Therapy, 2006, v. 13, n. 6, p. 1074, doi. 10.1016/j.ymthe.2006.03.005
By:
- Jacobson, Samuel G.;
- Acland, Gregory M.;
- Aguirre, Gustavo D.;
- Aleman, Tomas S.;
- Schwartz, Sharon B.;
- Cideciyan, Artur V.;
- Zeiss, Caroline J.;
- Komaromy, Andras M.;
- Kaushal, Shalesh;
- Roman, Alejandro J.;
- Windsor, Elizabeth A.M.;
- Sumaroka, Alexander;
- Pearce-Kelling, Susan E.;
- Conlon, Thomas J.;
- Chiodo, Vincent A.;
- Boye, Sanford L.;
- Flotte, Terence R.;
- Maguire, Albert M.;
- Bennett, Jean;
- Hauswirth, William W.
Publication type:
Article
Long-Term Restoration of Rod and Cone Vision by Single Dose rAAV-Mediated Gene Transfer to the Retina in a Canine Model of Childhood Blindness
Published in:
Molecular Therapy, 2005, v. 12, n. 6, p. 1072, doi. 10.1016/j.ymthe.2005.08.008
By:
- Acland, Gregory M.;
- Aguirre, Gustavo D.;
- Bennett, Jean;
- Aleman, Tomas S.;
- Cideciyan, Artur V.;
- Bennicelli, Jeannette;
- Dejneka, Nadine S.;
- Pearce-Kelling, Susan E.;
- Maguire, Albert M.;
- Palczewski, Krzysztof;
- Hauswirth, William W.;
- Jacobson, Samuel G.
Publication type:
Article
Systemic but not intraocular Epo Gene Transfer Protects the Retina from Light-and Genetic-Induced Degeneration
Published in:
Molecular Therapy, 2004, v. 10, n. 5, p. 855, doi. 10.1016/j.ymthe.2004.07.027
By:
- Rex, Tonia S.;
- Allocca, Mariacarmela;
- Domenici, Luciano;
- Surace, Enrico M.;
- Maguire, Albert M.;
- Lyubarsky, Arkady;
- Cellerino, Alessandro;
- Bennett, Jean;
- Auricchio, Alberto
Publication type:
Article
In Utero Gene Therapy Rescues Vision in a Murine Model of Congenital Blindness
Published in:
Molecular Therapy, 2004, v. 9, n. 2, p. 182, doi. 10.1016/j.ymthe.2003.11.013
By:
- Dejneka, Nadine S.;
- Surace, Enrico M.;
- Aleman, Tomas S.;
- Cideciyan, Artur V.;
- Lyubarsky, Arkady;
- Savchenko, Andrey;
- Redmond, T. Michael;
- Tang, Waixing;
- Wei, Zhangyong;
- Rex, Tonia S.;
- Glover, Ernest;
- Maguire, Albert M.;
- Pugh Jr., Edward N.;
- Jacobson, Samuel G.;
- Bennett, Jean
Publication type:
Article
Inhibition of Retinal Neovascularization by Intraocular Viral-Mediated Delivery of Anti-angiogenic Agents
Published in:
Molecular Therapy, 2002, v. 6, n. 4, p. 490, doi. 10.1006/mthe.2002.0702
By:
- Auricchio, Alberto;
- Behling, Kathryn C.;
- Maguire, Albert M.;
- O'Connor, Erin E.;
- Bennett, Jean;
- Wilson, James M.;
- Tolentino, Michael J.
Publication type:
Article
Pharmacological Regulation of Protein Expression from Adeno-Associated Viral Vectors in the Eye
Published in:
Molecular Therapy, 2002, v. 6, n. 2, p. 238, doi. 10.1006/mthe.2002.0660
By:
- Auricchio, Alberto;
- Rivera, Victor M.;
- Clackson, Tim;
- O'Connor, Erin E.;
- Maguire, Albert M.;
- Tolentino, Michael J.;
- Bennett, Jean;
- Wilson, James M.
Publication type:
Article
A Deviant Immune Response to Viral Proteins and Transgene Product Is Generated on Subretinal Administration of Adenovirus and Adeno-associated Virus
Published in:
Molecular Therapy, 2002, v. 5, n. 2, p. 125, doi. 10.1006/mthe.2002.0525
By:
- Anand, Vibha;
- Duffy, Bethany;
- Yang, Zaixin;
- Dejneka, Nadine S.;
- Maguire, Albert M.;
- Bennett, Jean
Publication type:
Article
A Virtual Reality Orientation and Mobility Test for Inherited Retinal Degenerations: Testing a Proof-of-Concept After Gene Therapy.
Published in:
Clinical Ophthalmology, 2021, v. 15, p. 939, doi. 10.2147/OPTH.S292527
By:
- Aleman, Tomas S;
- Miller, Alexander J;
- Maguire, Katherine H;
- Aleman, Elena M;
- Serrano, Leona W;
- O'Connor, Keli B;
- Bedoukian, Emma C;
- Leroy, Bart P;
- Maguire, Albert M;
- Bennett, Jean
Publication type:
Article
Mutations in ABCA4 result in accumulation of lipofuscin before slowing of the retinoid cycle: a reappraisal of the human disease sequence.
Published in:
Human Molecular Genetics, 2004, v. 13, n. 5, p. 525, doi. 10.1093/hmg/ddh048
By:
- Cideciyan, Artur V.;
- Aleman, Tomas S.;
- Swider, Malgorzata;
- Schwartz, Sharon B.;
- Steinberg, Janet D.;
- Brucker, Alexander J.;
- Maguire, Albert M.;
- Bennett, Jean;
- Stone, Edwin M.;
- Jacobson, Samuel G.
Publication type:
Article
Exchange of surface proteinsimpacts on viral vector cellular specificity and transduction characteristics: the retina as a model.
Published in:
Human Molecular Genetics, 2001, v. 10, n. 26, p. 3075, doi. 10.1093/hmg/10.26.3075
By:
- Auricchio, Alberto;
- Kobinger, Gary;
- Anand, Vibha;
- Hildinger, Markus;
- Connor, ErinO’;
- Maguire, Albert M.;
- Wilson, JamesM.;
- Bennett, Jean
Publication type:
Article

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