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References (122)
-
A. Auricchio, G. Kobinger, V. Anand, M. Hildinger, Erin O'Connor, A. Maguire, James Wilson, J. Bennett (2001)
Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model.Human molecular genetics, 10 26
-
T. Conlon, W. Deng, Kirsten Erger, Travis Cossette, J. Pang, R. Ryals, N. Clément, B. Cleaver, I. Mcdoom, S. Boye, M. Peden, M. Sherwood, C. Abernathy, F. Alkuraya, S. Boye, W. Hauswirth (2013)
Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa.Human gene therapy. Clinical development, 24 1
-
(2011)
Dosage thresholds and immune reactions to adeno-associated viruses need to be better evaluated to alleviate adverse effects in primates that have more sophisticated immune systems -
Adenovirus, lentivirus, or adeno-associated virus (AAV) can be used to transduce retinal pigment epithelium cells -
T. Cronin, L. Vandenberghe, Péter Hantz, Josephine Juttner, A. Reimann, Ágota Kacsó, R. Huckfeldt, V. Busskamp, H. Kohler, P. Lagali, B. Roska, J. Bennett (2014)
Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno-associated virus capsid and promoterEMBO Molecular Medicine, 6
-
J. Tang, T. Szikra, Y. Kozorovitskiy, M. Teixiera, B. Sabatini, B. Roska, C. Cepko (2013)
A Nanobody-Based System Using Fluorescent Proteins as Scaffolds for Cell-Specific Gene ManipulationCell, 154
-
L. Byrne, Yvonne Lin, Trevor Lee, D. Schaffer, J. Flannery (2015)
The expression pattern of systemically injected AAV9 in the developing mouse retina is determined by age.Molecular therapy : the journal of the American Society of Gene Therapy, 23 2
-
L. Dudus, V. Anand, G. Acland, Shu-Jen Chen, James Wilson, K. Fisher, A. Maguire, J. Bennett (1999)
Persistent transgene product in retina, optic nerve and brain after intraocular injection of rAAVVision Research, 39
-
Pavitra Ramachandran, V. Lee, Zhangyong Wei, Ji Song, G. Casal, T. Cronin, K. Willett, R. Huckfeldt, J. Morgan, T. Aleman, A. Maguire, J. Bennett (2017)
Evaluation of Dose and Safety of AAV7m8 and AAV8BP2 in the Non-Human Primate Retina.Human gene therapy, 28 2
-
Rangeetha Naik, A. Mukhopadhyay, M. Ganguli (2009)
Gene delivery to the retina: focus on non-viral approaches.Drug discovery today, 14 5-6
-
John Guy, X. Qi, Hui Wang, William Hauswirth (1999)
Adenoviral gene therapy with catalase suppresses experimental optic neuritis.Archives of ophthalmology, 117 11
-
M. Tan, Alexander Smith, B. Pawlyk, Xiaoyun Xu, Xiaoqing Liu, J. Bainbridge, M. Basche, J. McIntosh, H. Tran, A. Nathwani, Tiansen Li, R. Ali (2009)
Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors.Human molecular genetics, 18 12
-
AAV-mediated transgene expression has species-specific attributes -
Q. Lu, TH Ganjawala, E. Ivanova, Jg Cheng, D. Troilo, Z. Pan (2016)
AAV-Mediated Transduction and Targeting of Retinal Bipolar Cells with Improved mGluR6 Promoters in Rodents and PrimatesGene therapy, 23
-
N. Heintz (2001)
Bac to the future: The use of bac transgenic mice for neuroscience researchNature Reviews Neuroscience, 2
-
E. Sasaki, H. Suemizu, A. Shimada, K. Hanazawa, Ryo Oiwa, Michiko Kamioka, I. Tomioka, Y. Sotomaru, Reiko Hirakawa, T. Eto, Seiji Shiozawa, Takuji Maeda, Mamoru Ito, R. Ito, Chika Kito, Chie Yagihashi, K. Kawai, H. Miyoshi, Y. Tanioka, N. Tamaoki, S. Habu, H. Okano, T. Nomura (2009)
Generation of transgenic non-human primates with germline transmissionNature, 459
-
Michael Castle, H. Turunen, L. Vandenberghe, J. Wolfe (2016)
Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids.Methods in molecular biology, 1382
-
Junhuang Zou, Ling Luo, Zuolian Shen, V. Chiodo, B. Ambati, W. Hauswirth, Jun Yang (2011)
Whirlin replacement restores the formation of the USH2 protein complex in whirlin knockout photoreceptors.Investigative ophthalmology & visual science, 52 5
-
J. Belmonte, E. Callaway, S. Caddick, P. Churchland, G. Feng, G. Feng, G. Homanics, Kuo-Fen Lee, D. Leopold, Cory Miller, Jude Mitchell, S. Mitalipov, S. Mitalipov, Alysson Moutri, J. Movshon, H. Okano, J. Reynolds, D. Ringach, T. Sejnowski, T. Sejnowski, Afonso Silva, P. Strick, Jun Wu, Feng Zhang (2015)
Brains, Genes, and PrimatesNeuron, 86
-
G. Puras, J. Zárate, J. Zárate, Ariadna Díaz-Tahoces, M. Avilés-Trigueros, E. Fernández, J. Pedraz, J. Pedraz (2013)
Oligochitosan polyplexes as carriers for retinal gene delivery.European journal of pharmaceutical sciences : official journal of the European Federation for Pharmaceutical Sciences, 48 1-2
-
I. Trapani, A. Puppo, A. Auricchio (2014)
Vector platforms for gene therapy of inherited retinopathiesProgress in Retinal and Eye Research, 43
-
R. Dum, P. Strick (2013)
Transneuronal tracing with neurotropic viruses reveals network macroarchitectureCurrent Opinion in Neurobiology, 23
-
Viral vectors are more efficient for long-lasting, high-level transgene expression -
R. Farjo, J. Skaggs, A. Quiambao, M. Cooper, M. Naash (2006)
Efficient Non-Viral Ocular Gene Transfer with Compacted DNA NanoparticlesPLoS ONE, 1
-
D. Lipinski, M. Thake, R. MacLaren (2013)
Clinical applications of retinal gene therapyProgress in Retinal and Eye Research, 32
-
AAV is the most robust gene delivery vector in the neural retina -
Adam Packer, B. Roska, M. Häusser (2013)
Targeting neurons and photons for optogeneticsNature Neuroscience, 16
-
D. Dalkara, L. Byrne, R. Klimczak, Meike Visel, L. Yin, W. Merigan, J. Flannery, D. Schaffer (2013)
In Vivo–Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the VitreousScience Translational Medicine, 5
-
D. Biase, Sullivan, Wheeler, S. Brown, M. Myers, M. Mott, Vik, M. Cady, K. Winters, Jordan, D. Solberg, Stinchfield, R. Catalano, E. Wells, J. Jenson, Hawkins, J. Aftercare, De Leon, G. Melnick, Tims, J. Gans, Gaveras, L. America, Godley, R. Hubbard, E. Cavanaugh, S. Craddock, Rachal, R. Hawkins, Catalano, G. Joe, K. Broome, Rowan-Szal, Simpson, Broome, S. Maisto, T. Chung, J. Cornelius, Martin, C. Factor (2009)
Literature citedWetlands, 9
-
S. Boye, J. Alexander, C. Witherspoon, S. Boye, James Peterson, Mark Clark, Kristen Sandefer, C. Girkin, W. Hauswirth, Paul Gamlin (2016)
Highly Efficient Delivery of Adeno-Associated Viral Vectors to the Primate Retina.Human gene therapy, 27 8
-
A. Komáromy, J. Alexander, Jessica Rowlan, Monique Garcia, V. Chiodo, Asli Kaya, J. Tanaka, G. Acland, W. Hauswirth, G. Aguirre (2010)
Gene therapy rescues cone function in congenital achromatopsia.Human molecular genetics, 19 13
-
J. Alexander, W. Hauswirth (2008)
Adeno-associated viral vectors and the retina.Advances in experimental medicine and biology, 613
-
Benjamin Gaub, Michael Berry, Amy Holt, E. Isacoff, J. Flannery (2015)
Optogenetic Vision Restoration Using Rhodopsin for Enhanced SensitivityMolecular Therapy, 23
-
A. Manfredi, E. Marrocco, A. Puppo, G. Cesi, A. Sommella, M. Corte, S. Rossi, M. Giunti, C. Craft, M. Bacci, F. Simonelli, E. Surace, A. Auricchio (2013)
Combined rod and cone transduction by adeno-associated virus 2/8.Human gene therapy, 24 12
-
(2009)
Cre transgenicmouse lines . In Transgenesis Techniques : Principles and Protocols , ed . EJCartwright , pp . 265 – 73 . Meth . Mol . Biol . 561 -
K. Willett, J. Bennett (2013)
Immunology of AAV-Mediated Gene Transfer in the EyeFrontiers in Immunology, 4
-
T. Maclachlan, M. Lukason, Margaret Collins, R. Munger, Elisabete Isenberger, C. Rogers, S. Malatos, Elizabeth Dufresne, James Morris, R. Calcedo, G. Veres, A. Scaria, L. Andrews, S. Wadsworth (2011)
Preclinical safety evaluation of AAV2-sFLT01- a gene therapy for age-related macular degeneration.Molecular therapy : the journal of the American Society of Gene Therapy, 19 2
-
L. Byrne, D. Dalkara, G. Luna, S. Fisher, E. Clérin, J. Sahel, T. Léveillard, J. Flannery (2015)
Viral-mediated RdCVF and RdCVFL expression protects cone and rod photoreceptors in retinal degeneration.The Journal of clinical investigation, 125 1
-
Keisuke Yonehara, K. Farrow, Alexander Ghanem, D. Hillier, Kamill Bálint, Miguel Teixeira, J. Jüttner, M. Noda, R. Neve, K. Conzelmann, B. Roska (2013)
The First Stage of Cardinal Direction Selectivity Is Localized to the Dendrites of Retinal Ganglion CellsNeuron, 79
-
M. Inatani, H. Tanihara (2002)
Proteoglycans in retinaProgress in Retinal and Eye Research, 21
-
S. Boye, S. Boye, J. Pang, R. Ryals, D. Everhart, Y. Umino, A. Neeley, J. Besharse, R. Barlow, W. Hauswirth (2010)
Functional and Behavioral Restoration of Vision by Gene Therapy in the Guanylate Cyclase-1 (GC1) Knockout MousePLoS ONE, 5
-
M. Allocca, C. Mussolino, M. García-Hoyos, Daniela Sanges, C. Iodice, M. Petrillo, L. Vandenberghe, James Wilson, V. Marigo, E. Surace, A. Auricchio (2007)
Novel Adeno-Associated Virus Serotypes Efficiently Transduce Murine PhotoreceptorsJournal of Virology, 81
-
Adarsha Koirala, R. Makkia, Shannon Conley, M. Cooper, M. Naash (2013)
S/MAR-containing DNA nanoparticles promote persistent RPE gene expression and improvement in RPE65-associated LCA.Human molecular genetics, 22 8
-
L. Glushakova, A. Timmers, J. Pang, J. Teusner, W. Hauswirth (2006)
Human blue-opsin promoter preferentially targets reporter gene expression to rat s-cone photoreceptors.Investigative ophthalmology & visual science, 47 8
-
Transgene expression patterns are dictated by delivery route, AAV capsid, state of the retina, viral dose, and promoter -
Zongchao Han, Shannon Conley, R. Makkia, Junjing Guo, M. Cooper, M. Naash (2012)
Comparative Analysis of DNA Nanoparticles and AAVs for Ocular Gene DeliveryPLoS ONE, 7
-
V. Busskamp, Jens Duebel, D. Bálya, Mathias Fradot, T. Viney, S. Siegert, A. Groner, E. Cabuy, V. Forster, M. Seeliger, M. Biel, P. Humphries, M. Pâques, S. Mohand-Said, D. Trono, K. Deisseroth, J. Sahel, S. Picaud, B. Roska (2010)
Genetic Reactivation of Cone Photoreceptors Restores Visual Responses in Retinitis PigmentosaScience, 329
-
Dana Lau, Laura Mcgee, Shangzhen Zhou, Katherine Rendahl, William Manning, Jaime Escobedo, John Flannery (2000)
Retinal degeneration is slowed in transgenic rats by AAV-mediated delivery of FGF-2.Investigative ophthalmology & visual science, 41 11
-
F. Dyka, S. Boye, R. Ryals, V. Chiodo, S. Boye, W. Hauswirth (2014)
Cone specific promoter for use in gene therapy of retinal degenerative diseases.Advances in experimental medicine and biology, 801
-
M. Hellström, M. Ruitenberg, M. Pollett, E. Ehlert, J. Twisk, J. Verhaagen, A. Harvey (2009)
Cellular tropism and transduction properties of seven adeno-associated viral vector serotypes in adult retina after intravitreal injectionGene Therapy, 16
-
H. Petrs-Silva, A. Dinculescu, Qiuhong Li, W. Deng, J. Pang, Seok-Hong Min, V. Chiodo, A. Neeley, L. Govindasamy, A. Bennett, M. Agbandje-McKenna, Li Zhong, Baozheng Li, G. Jayandharan, A. Srivastava, A. Lewin, W. Hauswirth (2011)
Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina.Molecular therapy : the journal of the American Society of Gene Therapy, 19 2
-
Brian Marsumoro, J. Blanks, S. Ryan (1984)
Topographic variations in the rabbit and primate internal limiting membrane.Investigative ophthalmology & visual science, 25 1
-
I. Trapani, P. Colella, A. Sommella, C. Iodice, G. Cesi, Sonia Simone, E. Marrocco, S. Rossi, M. Giunti, A. Palfi, G. Farrar, R. Polishchuk, A. Auricchio (2013)
Effective delivery of large genes to the retina by dual AAV vectorsEMBO Molecular Medicine, 6
-
Natalia Caporale, K. Kolstad, Trevor Lee, Ivan Tochitsky, D. Dalkara, D. Trauner, R. Kramer, Y. Dan, E. Isacoff, J. Flannery (2011)
LiGluR restores visual responses in rodent models of inherited blindness.Molecular therapy : the journal of the American Society of Gene Therapy, 19 7
-
G. Acland, G. Aguirre, J. Bennett, T. Aleman, A. Cideciyan, J. Bennicelli, N. Dejneka, S. Pearce-Kelling, A. Maguire, K. Palczewski, W. Hauswirth, S. Jacobson (2005)
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.Molecular therapy : the journal of the American Society of Gene Therapy, 12 6
-
P. Apaolaza, A. Pozo-Rodríguez, M. Solinís, J. Rodríguez, U. Friedrich, J. Torrecilla, B. Weber, A. Rodríguez-Gascón (2016)
Structural recovery of the retina in a retinoschisin-deficient mouse after gene replacement therapy by solid lipid nanoparticles.Biomaterials, 90
-
D. Dalkara, K. Kolstad, Natalia Caporale, Meike Visel, R. Klimczak, D. Schaffer, J. Flannery (2009)
Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous.Molecular therapy : the journal of the American Society of Gene Therapy, 17 12
-
Yasmine El-Shamayleh, Amy Ni, G. Horwitz (2016)
Strategies for targeting primate neural circuits with viral vectors.Journal of neurophysiology, 116 1
-
J. Flannery, S. Zolotukhin, M. Vaquero, M. Lavail, N. Muzyczka, W. Hauswirth (1997)
Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus.Proceedings of the National Academy of Sciences of the United States of America, 94 13
-
C. Kay, R. Ryals, G. Aslanidi, Seok-Hong Min, Qing Ruan, Jingfen Sun, F. Dyka, Daniel Kasuga, Andrea Ayala, Kim Vliet, M. Agbandje-McKenna, W. Hauswirth, S. Boye, S. Boye (2013)
Targeting Photoreceptors via Intravitreal Delivery Using Novel, Capsid-Mutated AAV VectorsPLoS ONE, 8
-
Michael Dorrell, E. Aguilar, R. Jacobson, Ó. Yanes, R. Gariano, J. Heckenlively, E. Banin, G. Ramirez, M. Gasmi, A. Bird, G. Siuzdak, M. Friedlander (2009)
Antioxidant or neurotrophic factor treatment preserves function in a mouse model of neovascularization-associated oxidative stress.The Journal of clinical investigation, 119 3
-
F. Mazzoni, Hussein Safa, S. Finnemann (2014)
Understanding photoreceptor outer segment phagocytosis: use and utility of RPE cells in culture.Experimental eye research, 126
-
S. Boye, J. Alexander, S. Boye, C. Witherspoon, Kristen Sandefer, T. Conlon, Kirsten Erger, Jingfen Sun, R. Ryals, V. Chiodo, Mark Clark, C. Girkin, W. Hauswirth, Paul Gamlin (2012)
The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina.Human gene therapy, 23 10
-
Inchan Kwon, D. Schaffer (2007)
Designer Gene Delivery Vectors: Molecular Engineering and Evolution of Adeno-Associated Viral Vectors for Enhanced Gene TransferPharmaceutical Research, 25
-
C. Mussolino, M. Corte, S. Rossi, F. Viola, U. Vicino, E. Marrocco, S. Neglia, M. Dòria, F. Testa, R. Giovannoni, M. Crasta, M. Giunti, E. Villani, M. Lavitrano, M. Bacci, R. Ratiglia, F. Simonelli, A. Auricchio, E. Surace (2011)
AAV-mediated photoreceptor transduction of the pig cone-enriched retinaGene Therapy, 18
-
C. Cearley, J. Wolfe (2006)
Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain.Molecular therapy : the journal of the American Society of Gene Therapy, 13 3
-
Jianwen Liu, A. Timmers, A. Lewin, W. Hauswirth (2005)
Ribozyme Knockdown of the γ-Subunit of Rod cGMP Phosphodiesterase Alters the ERG and Retinal Morphology in Wild-Type MiceInvestigative Ophthalmology & Visual Science, 46
-
D. Tervo, B. Hwang, Sarada Viswanathan, T. Gaj, Maria Lavzin, Maria Lavzin, K. Ritola, S. Lindo, Susan Michael, Elena Kuleshova, Elena Kuleshova, David Ojala, Cheng-Chiu Huang, C. Gerfen, J. Schiller, J. Schiller, J. Dudman, A. Hantman, L. Looger, D. Schaffer, A. Karpova (2016)
A Designer AAV Variant Permits Efficient Retrograde Access to Projection NeuronsNeuron, 92
-
J. Koerber, R. Klimczak, Jae‐Hyung Jang, D. Dalkara, J. Flannery, D. Schaffer (2009)
Molecular evolution of adeno-associated virus for enhanced glial gene delivery.Molecular therapy : the journal of the American Society of Gene Therapy, 17 12
-
T. Park, Zhijian Wu, S. Kjellstrom, Yong Zeng, R. Bush, P. Sieving, P. Colosi (2009)
Intravitreal delivery of AAV8 retinoschisin results in cell type-specific gene expression and retinal rescue in the Rs1-KO mouseGene Therapy, 16
-
Susan Shen, C. Myers, Andrew Hughes, L. Byrne, J. Flannery, J. Corbo (2016)
Massively parallel cis-regulatory analysis in the mammalian central nervous systemGenome Research, 26
-
H. Petrs-Silva, A. Dinculescu, Qiuhong Li, Seok-Hong Min, V. Chiodo, J. Pang, Li Zhong, S. Zolotukhin, A. Srivastava, A. Lewin, W. Hauswirth (2009)
High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors.Molecular therapy : the journal of the American Society of Gene Therapy, 17 3
-
Kizito-Tshitoko Tshilenge, B. Ameline, M. Weber, A. Mendes-Madeira, S. Nedellec, M. Biget, N. Provost, L. Libeau, V. Blouin, J. Deschamps, G. Meur, M. Colle, P. Moullier, V. Pichard, F. Rolling (2016)
Vitrectomy Before Intravitreal Injection of AAV2/2 Vector Promotes Efficient Transduction of Retinal Ganglion Cells in Dogs and Nonhuman Primates.Human gene therapy methods, 27 3
-
E. Sutanto, Dan Zhang, Y. Lai, W. Shen, E. Rakoczy (2005)
Development and Evaluation of the Specificity of a Cathepsin D Proximal Promoter in the EyeCurrent Eye Research, 30
-
G. Ye, E. Budzynski, P. Sonnentag, T. Nork, N. Sheibani, Z. Gurel, S. Boye, James Peterson, S. Boye, W. Hauswirth, J. Chulay (2016)
Cone-Specific Promoters for Gene Therapy of Achromatopsia and Other Retinal Diseases.Human gene therapy, 27 1
-
Xin Wang (2009)
Cre transgenic mouse lines.Methods in molecular biology, 561
-
(2016)
Comander et al. 2016) will need to be developed to obtain better gene delivery and cell specificity in the primate retina -
J. Adijanto, M. Naash (2015)
Nanoparticle-based technologies for retinal gene therapy.European journal of pharmaceutics and biopharmaceutics : official journal of Arbeitsgemeinschaft fur Pharmazeutische Verfahrenstechnik e.V, 95 Pt B
-
Lucie Pellissier, Peter Quinn, C. Alves, R. Vos, J. Klooster, John Flannery, J. Heimel, J. Wijnholds (2015)
Gene therapy into photoreceptors and Müller glial cells restores retinal structure and function in CRB1 retinitis pigmentosa mouse models.Human molecular genetics, 24 11
-
K. Stieger, Elsa Lhériteau, P. Moullier, F. Rolling (2009)
AAV-mediated gene therapy for retinal disorders in large animal models.ILAR journal, 50 2
-
J. Comander, Livia Carvalho, Ru Xiao, Aliete Langsdorf, Sarah Wassmer, B. Hafler, David Wu, D. Eliott, L. Kim, L. Vandenberghe (2016)
Characterization of the limitations to gene transfer and associated inflammation following intravitreal AAV injection in nonhuman primatesInvestigative Ophthalmology & Visual Science, 57
-
S. Mckinnon, D. Lehman, N. Tahzib, N. Ransom, H. Reitsamer, P. Liston, E. LaCasse, Qiuhong Li, R. Korneluk, W. Hauswirth (2002)
Baculoviral IAP repeat-containing-4 protects optic nerve axons in a rat glaucoma model.Molecular therapy : the journal of the American Society of Gene Therapy, 5 6
-
V. Busskamp, B. Roska (2011)
Optogenetic approaches to restoring visual function in retinitis pigmentosaCurrent Opinion in Neurobiology, 21
-
M. Kotterman, L. Yin, J. Strazzeri, J. Flannery, W. Merigan, D. Schaffer (2014)
Antibody neutralization poses a barrier to intravitreal adeno-associated viral vector gene delivery to non-human primatesGene Therapy, 22
-
C. Leeuw, F. Dyka, S. Boye, Stéphanie Laprise, Michelle Zhou, A. Chou, Lisa Borretta, Simone McInerny, K. Banks, E. Portales-Casamar, Magdalena Swanson, C. D'souza, S. Boye, Steven Jones, R. Holt, D. Goldowitz, W. Hauswirth, W. Wasserman, E. Simpson (2014)
Targeted CNS delivery using human MiniPromoters and demonstrated compatibility with adeno-associated viral vectorsMolecular Therapy. Methods & Clinical Development, 1
-
E. Ivanova, Grace-Soon Hwang, Z. Pan, D. Troilo (2010)
Evaluation of AAV-mediated expression of Chop2-GFP in the marmoset retina.Investigative ophthalmology & visual science, 51 10
-
P. Colella, I. Trapani, G. Cesi, A. Sommella, A. Manfredi, A. Puppo, C. Iodice, S. Rossi, F. Simonelli, M. Giunti, M. Bacci, A. Auricchio (2014)
Efficient gene delivery to the cone-enriched pig retina by dual AAV vectorsGene Therapy, 21
-
M. Kay, J. Glorioso, L. Naldini (2001)
Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeuticsNature Medicine, 7
-
Rajeshwari Koilkonda, J. Guy (2010)
Leber's Hereditary Optic Neuropathy-Gene Therapy: From Benchtop to BedsideJournal of Ophthalmology, 2011
-
John Lin, Michael Lin, Paul Steinbach, R. Tsien (2009)
Characterization of engineered channelrhodopsin variants with improved properties and kinetics.Biophysical journal, 96 5
-
J. Cehajic-Kapetanovic, M. Goff, A. Allen, R. Lucas, P. Bishop (2010)
Glycosidic enzymes enhance retinal transduction following intravitreal delivery of AAV2Molecular Vision, 17
-
M. Doroudchi, K. Greenberg, Jianwen Liu, Kimberly Silka, E. Boyden, Jennifer Lockridge, A. Arman, R. Janani, S. Boye, S. Boye, G. Gordon, Benjamin Matteo, A. Sampath, W. Hauswirth, Alan Horsager (2011)
Virally delivered channelrhodopsin-2 safely and effectively restores visual function in multiple mouse models of blindness.Molecular therapy : the journal of the American Society of Gene Therapy, 19 7
-
A. Huberman, Mihai Manu, Selina Koch, Michael Susman, A. Lutz, E. Ullian, S. Baccus, B. Barres (2008)
Architecture and Activity-Mediated Refinement of Axonal Projections from a Mosaic of Genetically Identified Retinal Ganglion CellsNeuron, 59
-
J. Alexander, Y. Umino, D. Everhart, B. Chang, Seok-Hong Min, Qiuhong Li, A. Timmers, N. Hawes, J. Pang, R. Barlow, W. Hauswirth (2007)
Restoration of cone vision in a mouse model of achromatopsiaNature Medicine, 13
-
Qiuhong Li, A. Timmers, J. Guy, J. Pang, W. Hauswirth (2008)
Cone-specific expression using a human red opsin promoter in recombinant AAVVision Research, 48
-
R. Mitra, Zongchao Han, Miles Merwin, Muhammed Al Taai, Shannon Conley, M. Naash (2014)
Synthesis and Characterization of Glycol Chitosan DNA Nanoparticles for Retinal Gene DeliveryChemMedChem, 9
-
Emilie Mace, R. Caplette, O. Marre, Abhishek Sengupta, A. Chaffiol, Peggy Barbe, M. Desrosiers, E. Bamberg, J. Sahel, S. Picaud, Jens Duebel, D. Dalkara (2015)
Targeting channelrhodopsin-2 to ON-bipolar cells with vitreally administered AAV Restores ON and OFF visual responses in blind mice.Molecular therapy : the journal of the American Society of Gene Therapy, 23 1
-
research will focus on the nonhuman primate retina to get closer to the human retina for both basic science and gene therapy applications -
Miranda Scalabrino, S. Boye, K. Fransen, Jennifer Noel, F. Dyka, Seok-Hong Min, Qing Ruan, C. Leeuw, E. Simpson, R. Gregg, M. McCall, N. Peachey, S. Boye (2015)
Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness.Human molecular genetics, 24 21
-
S. Khani, B. Pawlyk, O. Bulgakov, E. Kasperek, Joyce Young, M. Adamian, Xun Sun, Alexander Smith, R. Ali, Tiansen Li (2007)
AAV-mediated expression targeting of rod and cone photoreceptors with a human rhodopsin kinase promoter.Investigative ophthalmology & visual science, 48 9
-
TL Fink, PJ Klepcyk, SM Oette, CR Gedeon, SL Hyatt, TH Kowalczyk, RC Moen, MJ Cooper (2006)
Plasmid size up to 20 kbp does not limit effective in vivo lung gene transfer using compacted DNA nanoparticlesGene Therapy, 13
-
K. Guziewicz, B. Zangerl, A. Komáromy, S. Iwabe, V. Chiodo, S. Boye, W. Hauswirth, W. Beltran, G. Aguirre (2013)
Recombinant AAV-Mediated BEST1 Transfer to the Retinal Pigment Epithelium: Analysis of Serotype-Dependent Retinal EffectsPLoS ONE, 8
-
Zongchao Han, Shannon Conley, R. Makkia, M. Cooper, M. Naash (2012)
DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice.The Journal of clinical investigation, 122 9
-
L. Vandenberghe, Peter Bell, A. Maguire, Ru Xiao, T. Hopkins, R. Grant, J. Bennett, James Wilson (2013)
AAV9 Targets Cone Photoreceptors in the Nonhuman Primate RetinaPLoS ONE, 8
-
E. Callaway (2008)
Transneuronal circuit tracing with neurotropic virusesCurrent Opinion in Neurobiology, 18
-
D. Dalkara, L. Byrne, Trevor Lee, N. Hoffmann, David Schaffer, John Flannery (2011)
Enhanced gene delivery to the neonatal retina through systemic administration of tyrosine-mutated AAV9Gene Therapy, 19
-
A. Auricchio, K. Behling, A. Maguire, Erin O'Connor, J. Bennett, James Wilson, M. Tolentino (2002)
Inhibition of retinal neovascularization by intraocular viral-mediated delivery of anti-angiogenic agents.Molecular therapy : the journal of the American Society of Gene Therapy, 6 4
-
Brian Zingg, Xiao-lin Chou, Zheng-gang Zhang, Lukas Mesik, F. Liang, H. Tao, Li Zhang (2017)
AAV-Mediated Anterograde Transsynaptic Tagging: Mapping Corticocollicular Input-Defined Neural Pathways for Defense BehaviorsNeuron, 93
-
K. Kolstad, D. Dalkara, K. Guerin, Meike Visel, N. Hoffmann, D. Schaffer, J. Flannery (2010)
Changes in adeno-associated virus-mediated gene delivery in retinal degeneration.Human gene therapy, 21 5
-
R. Klimczak, J. Koerber, D. Dalkara, J. Flannery, D. Schaffer (2009)
A Novel Adeno-Associated Viral Variant for Efficient and Selective Intravitreal Transduction of Rat Müller CellsPLoS ONE, 4
-
L. Yin, K. Greenberg, Jennifer Hunter, D. Dalkara, K. Kolstad, Benjamin Masella, R. Wolfe, Meike Visel, D. Stone, R. Libby, D. Diloreto, D. Schaffer, J. Flannery, David Williams, W. Merigan (2011)
Intravitreal injection of AAV2 transduces macaque inner retina.Investigative ophthalmology & visual science, 52 5
-
S. Michalakis, R. Mühlfriedel, N. Tanimoto, Vidhyasankar Krishnamoorthy, S. Koch, M. Fischer, E. Becirovic, Lin Bai, G. Huber, Susanne Beck, E. Fahl, H. Büning, François Paquet-Durand, X. Zong, T. Gollisch, M. Biel, M. Seeliger (2010)
Restoration of cone vision in the CNGA3-/- mouse model of congenital complete lack of cone photoreceptor function.Molecular therapy : the journal of the American Society of Gene Therapy, 18 12
-
Andrea Granstedt, Moriah Szpara, B. Kuhn, S. Wang, L. Enquist (2009)
Fluorescence-Based Monitoring of In Vivo Neural Activity Using a Circuit-Tracing Pseudorabies VirusPLoS ONE, 4
-
Gregory Wojaczynski, E. Engel, Karina Steren, L. Enquist, bullet Card, Ó. Springer-Verlag, Berlin Heidelberg (2014)
The neuroinvasive profiles of H129 (herpes simplex virus type 1) recombinants with putative anterograde-only transneuronal spread propertiesBrain Structure and Function, 220
-
A. Huberman, Wei Wei, J. Elstrott, Benjamin Stafford, M. Feller, B. Barres (2009)
Genetic Identification of an On-Off Direction- Selective Retinal Ganglion Cell Subtype Reveals a Layer-Specific Subcortical Map of Posterior MotionNeuron, 62
-
Katherine Mancuso, W. Hauswirth, Qiuhong Li, T. Connor, J. Kuchenbecker, M. Mauck, J. Neitz, M. Neitz (2009)
Gene therapy for red-green colour blindness in adult primatesNature, 461
-
M. Kotterman, D. Schaffer (2014)
Engineering adeno-associated viruses for clinical gene therapyNature Reviews Genetics, 15
-
L. Battaglia, L. Serpe, F. Foglietta, E. Muntoni, M. Gallarate, Ana Rodríguez, M. Solinís (2016)
Application of lipid nanoparticles to ocular drug deliveryExpert Opinion on Drug Delivery, 13
-
L. Vandenberghe, Peter Bell, A. Maguire, C. Cearley, Ru Xiao, R. Calcedo, Lili Wang, Michael Castle, Alexandra Maguire, R. Grant, John Wolfe, James Wilson, Jean Bennett (2011)
Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in MonkeyScience Translational Medicine, 3
-
H. Prentice, M. Biswal, C. Dorey, J. Blanks (2011)
Hypoxia-regulated retinal glial cell-specific promoter for potential gene therapy in disease.Investigative ophthalmology & visual science, 52 12
-
G. Meur, K. Stieger, Alexander Smith, Michel Weber, J. Deschamps, D. Nivard, A. Mendes-Madeira, N. Provost, Y. Péréon, Y. Chérel, R. Ali, Christian Hamel, P. Moullier, F. Rolling (2007)
Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epitheliumGene Therapy, 14
-
J. Tang, Stephanie Rudolph, Onkar Dhande, V. Abraira, Seungwon Choi, S. Lapan, Iain Drew, Eugene Drokhlyansky, A. Huberman, W. Regehr, C. Cepko (2015)
Cell Type-Specific Manipulation with GFP-Dependent Cre RecombinaseNature neuroscience, 18
- Publisher
- Annual Reviews
- Copyright
- Copyright 2017 by Annual Reviews. All rights reserved
- ISSN
- 2374-4642
- eISSN
- 2374-4650
- DOI
- 10.1146/annurev-vision-102016-061413
- pmid
- 28937950
- Publisher site
- See Article on Publisher Site
Abstract
One of the great advantages of the retina as a target tissue for gene delivery is the wide array of genetic tools that have been developed in the past decade. This includes a variety of vectors for therapeutic gene delivery to most types of retinal neurons and glia, as well as cell typespecific promoters for restricted gene expression in distinct neuronal subtypes. Within the scope of neuroscience applications and for gene therapy, it is now routine to express reporter genes, replacement genes, neuronal activity indicators, and microbial opsins in specific neuronal types in the mouse retina. However, there are considerable anatomical, physiological, immunological, and behavioral differences between the mouse and the human that limit the usefulness of these tools in humans and nonhuman primates. Several advances are now being made toward the goal of applying viral targeting tools to understand the primate retina. Here, we describe these advances, consider their potential to advance our understanding of the primate retina, and describe what will be needed to move forward.
Journal
Annual review of vision science – Annual Reviews
Published: Sep 15, 2017