Research Topics
Genomes and Genes
| Jean BennettSummaryAffiliation: University of Pennsylvania Country: USA Publications
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Detail Information
Publications
AAV2 gene therapy readministration in three adults with congenital blindnessJean Bennett
F M Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, 309 Stellar Chance Labs, 422 Curie Boulevard, Philadelphia, PA 19104, USA
Sci Transl Med 4:120ra15. 2012....
Commentary: an aye for eye gene therapyJean Bennett
F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA 19104, USA
Hum Gene Ther 17:177-9. 2006
Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transferJeannette Bennicelli
Department of Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, Pennsylvania 19104 6069, USA
Mol Ther 16:458-65. 2008..The data demonstrate that AAV2.RPE65 delivers the RPE65 transgene efficiently and quickly to the appropriate target cells in vivo in animal models. This vector holds great promise for treatment of LCA due to RPE65 mutations...- Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retinaJ Bennett
Department of Ophthalmology, F M Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, 51 North 39th Street, Philadelphia, PA 19104, USA
Proc Natl Acad Sci U S A 96:9920-5. 1999..The findings further characterize the retinal cell tropism of rAAV. They also support the development of studies aimed ultimately at treating inherited retinal degeneration by using rAAV-mediated gene therapy... - Gene therapy for choroideremia: in vitro rescue mediated by recombinant adenovirusVibha Anand
Department of Ophthalmology, F M Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania School of Medicine, Philadelphia, PA 19104 6069, USA
Vision Res 43:919-26. 2003..Adenovirus-mediated delivery of REP-1 rescued the defective cells as assessed through protein and enzymatic assays. Ultimately, it may be possible to use virus-mediated delivery of REP-1 to evaluate disease intervention in vivo... - Gene therapy for retinitis pigmentosaJ Bennett
FM Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania School of Medicine, PA 19104 6069, USA
Curr Opin Mol Ther 2:420-5. 2000..While much more progress needs to be made before moving from the laboratory to the clinic, gene therapy now holds much promise for slowing or even preventing blindness due to RP... - In utero gene therapy rescues vision in a murine model of congenital blindnessNadine S Dejneka
F.M. Kirby Center and Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, 51 N. 39th Street, Philadelphia, PA 19104-2689, USA
Mol Ther 9:182-8. 2004..The results demonstrate AAV-mediated correction of the deficit and suggest that in utero retinal gene delivery may be a useful approach for treating a variety of blinding congenital retinal diseases... - Safety and efficacy of gene transfer for Leber's congenital amaurosisAlbert M Maguire
Scheie Eye Institute, University of Pennsylvania, USA
N Engl J Med 358:2240-8. 2008..Although the follow-up was very short and normal vision was not achieved, this study provides the basis for further gene therapy studies in patients with LCA... - Nonhuman primate models for diabetic ocular neovascularization using AAV2-mediated overexpression of vascular endothelial growth factorCorinna Lebherz
Department of Medical Genetics, University of Pennsylvania, Philadelphia, Pennsylvania 190104-6069, USA
Diabetes 54:1141-9. 2005..Nonhuman primate models will be useful in testing long-term safety and efficacy of novel therapeutic agents for blinding neovascular diseases... - Delivery of adeno-associated virus vectors to the fetal retina: impact of viral capsid proteins on retinal neuronal progenitor transductionEnrico M Surace
F. M. Kirby Center for Molecular Ophthalmology, Department of Ophthalmology, Scheie Eye Institute, 310 Stellar Chance Building, University of Pennsylvania, 422 Curie Boulevard, Philadelphia, PA 19104-6069, USA
J Virol 77:7957-63. 2003..The procedure is not detrimental to retinal development and function and therefore provides a safe delivery vehicle for potential therapeutic applications and a means of assessing the mechanisms of retina development and disease... - Long-term inducible gene expression in the eye via adeno-associated virus gene transfer in nonhuman primatesCorinna Lebherz
Gene Therapy Program, Department of Medicine, Division of Medical Genetics, University of Pennsylvania, Philadelphia, PA 19104, USA
Hum Gene Ther 16:178-86. 2005..5 years. The expression profiles were characterized by minimal basal expression in the absence of inducer and dose-responsive maximal expression in the presence of inducer drug... - Effect of over-expression of pigment epithelium derived factor (PEDF) on developing retinal vasculature in the mouseWai T Wong
F. M. Kirby Center for Molecular Ophthalmology, Department of Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia 19104-6069, USA
Mol Vis 10:837-44. 2004..The findings are relevant to the safety of the potential therapeutic use of pigment epithelium derived factor in human retinal disease... - Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trialAlbert M Maguire
F M Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA 19104, USA
Lancet 374:1597-605. 2009..We therefore did a phase 1 trial to assess the effect of gene therapy on retinal and visual function in children and adults with Leber's congenital amaurosis... - Systemic rapamycin inhibits retinal and choroidal neovascularization in miceNadine S Dejneka
F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA
Mol Vis 10:964-72. 2004..CONCLUSIONS: These results suggest rapamycin may provide an effective new treatment for ocular neovascularization... - Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindnessDefne Amado
F M Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA 19104, USA
Sci Transl Med 2:21ra16. 2010..Thus, subretinal readministration of rAAV2 in animals is safe and effective, even in the setting of preexisting immunity to the vector, a parameter that has been used to exclude patients from gene therapy trials... - Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy successSamuel G Jacobson
Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA 19104, USA
Proc Natl Acad Sci U S A 102:6177-82. 2005.... - The distribution, concentration, and toxicity of enhanced green fluorescent protein in retinal cells after genomic or somatic (virus-mediated) gene transferTonia S Rex
F M Kirby Center for Molecular Ophthalmology, University of Pennsylvania, Philadelphia, PA 19104, USA
Mol Vis 11:1236-45. 2005..The concentration of enhanced green fluorescent protein (EGFP) in individual photoreceptor cells of live mouse retina was quantified and correlated with physiological measurements of cell function... - Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injectionSamuel G Jacobson
Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA 19104, USA
Mol Ther 13:1074-84. 2006..5-log unit range of vector doses proved efficacious. The efficacy and toxicity limits defined in this study lead to suggestions for the design of a subretinal AAV-2/2.RPE65 human trial of RPE65-associated LCA... - Systemic but not intraocular Epo gene transfer protects the retina from light-and genetic-induced degenerationTonia S Rex
F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA 19104, USA
Mol Ther 10:855-61. 2004..In conclusion, our data support Epo as a neuroprotective agent in some, but not all, retinal degenerations. Further, rescue is observed in specific models after systemic but not intraocular Epo gene transfer... - The human visual cortex responds to gene therapy-mediated recovery of retinal functionManzar Ashtari
Diffusion Tensor Image Analyses and Brain Morphometry Center, Department of Radiology, Children s Hospital of Philadelphia CHOP, Philadelphia, Pennsylvania 19104, USA
J Clin Invest 121:2160-8. 2011..In addition, these data suggest that gene therapy resulted in not only sustained and improved visual ability, but also enhanced contrast sensitivity... - ABCA4-associated retinal degenerations spare structure and function of the human parapapillary retinaArtur V Cideciyan
Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA
Invest Ophthalmol Vis Sci 46:4739-46. 2005..To study the parapapillary retinal region in patients with ABCA4-associated retinal degenerations... - Light damage induced changes in mouse retinal gene expressionLin Chen
F.M. Kirby Center for Molecular Ophthalmology Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA 19104, USA
Exp Eye Res 79:239-47. 2004..The genes found upregulated by light damage may affect the survival of photoreceptors subjected to photo-oxidative stress... - Mutations in ABCA4 result in accumulation of lipofuscin before slowing of the retinoid cycle: a reappraisal of the human disease sequenceArtur V Cideciyan
Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA 19104, USA
Hum Mol Genet 13:525-34. 2004..Knowledge of the human ABCA4 disease sequence will be critical for defining rates of progression, selecting appropriate patients and retinal locations for future therapy, and choosing appropriate treatment outcomes... - Disease expression in Usher syndrome caused by VLGR1 gene mutation (USH2C) and comparison with USH2A phenotypeSharon B Schwartz
Department of Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA
Invest Ophthalmol Vis Sci 46:734-43. 2005..To investigate the retinal disease expression in USH2C, the subtype of Usher syndrome type 2 recently shown to be caused by mutation in the VLGR1 gene, and compare results with those from USH2A, a more common cause of Usher syndrome... - Novel AAV serotypes for improved ocular gene transferCorinna Lebherz
Gene Therapy Program, Division of Medical Genetics, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA
J Gene Med 10:375-82. 2008..We show here that AAV7 and 8 enable superior long-term transduction of retinal and also anterior chamber structures... - Persistent fetal ocular vasculature in mice deficient in bax and bakPaul Hahn
Departments of Medicine, Pathology, and Laboratory Medicine, Abramson Family Cancer Research Institute, University of Pennsylvania, Philadelphia, USA
Arch Ophthalmol 123:797-802. 2005..The ocular fetal vasculature normally regresses by apoptosis but for unknown reasons fails to regress in the human disease persistent fetal vasculature... - Gene transfer to ocular stem cells by early gestational intraamniotic injection of lentiviral vectorMasayuki Endo
The Children s Center for Fetal Research, Children s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA
Mol Ther 15:579-87. 2007..This model may be useful for the investigation of mechanisms of genetic and/or developmental ocular disease and for the development of prenatal gene therapy for specific ocular disorders... - Impairment of the transient pupillary light reflex in Rpe65(-/-) mice and humans with leber congenital amaurosisTomas S Aleman
Department of Ophthalmology, University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA
Invest Ophthalmol Vis Sci 45:1259-71. 2004..To determine the impairment of the transient pupillary light reflex (TPLR) due to severe retinal dysfunction and degeneration in a murine model of Leber congenital amaurosis (LCA) and in patients with the disease... - Spatial and temporal expression patterns of the choroideremia gene in the mouse retinaNicholas W Keiser
Department of Ophthalmology, F M Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA 19104 6069, USA
Mol Vis 11:1052-60. 2005..A study of the expression patterns of Chm and the related gene Chml in the mouse retina was undertaken in order to address this issue... - Proapoptotic bcl-2 family members, Bax and Bak, are essential for developmental photoreceptor apoptosisPaul Hahn
F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, Philadelphia, Pennsylvania, USA
Invest Ophthalmol Vis Sci 44:3598-605. 2003..Future studies will investigate the potential role of Bax and Bak in mediating pathologic photoreceptor death... - Small interfering RNA (siRNA) targeting VEGF effectively inhibits ocular neovascularization in a mouse modelSamuel J Reich
F. M. Kirby Center for Molecular Ophthalmology, Department of Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia 19104, USA
Mol Vis 9:210-6. 2003..This work suggests that RNA interference has potential for application to studies of retinal biology and for the treatment of a variety of retinal diseases, including those involving abnormal blood vessel growth... - Inhibition of retinal neovascularization by intraocular viral-mediated delivery of anti-angiogenic agentsAlberto Auricchio
F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA
Mol Ther 6:490-4. 2002..All three vectors inhibited ischemia-induced neovascularization... - AAV-mediated delivery of ciliary neurotrophic factor prolongs photoreceptor survival in the rhodopsin knockout mouseF Q Liang
F. M. Kirby Center for Molecular Ophthalmology, University of Pennsylvania, Philadelphia, Pennsylvania, 19104, USA
Mol Ther 3:241-8. 2001..This protection lasted through termination of the experiment (3 months). AAV-mediated delivery of CNTF may have implications for the treatment of human retinal degeneration... - Phenotypic rescue after adeno-associated virus-mediated delivery of 4-sulfatase to the retinal pigment epithelium of feline mucopolysaccharidosis VIThucanh T Ho
F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA
J Gene Med 4:613-21. 2002.... - Adeno-associated viruses containing bFGF or BDNF are neuroprotective against excitotoxicityFrank Schuettauf
Department of Ophthalmology, University of Pennsylvania, Scheie Eye Institute, Philadelphia, Pennsylvania, USA
Curr Eye Res 29:379-86. 2004..We tested the possibility of delivering these growth factors to retinal ganglion cells via an adeno-associated viral (AAV) vector and tested their efficacy in two models of excitotoxicity... - In vivo delivery of recombinant viruses to the fetal murine cochlea: transduction characteristics and long-term effects on auditory functionJeffrey C Bedrosian
FM Kirby Center and Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania School of Medicine, Philadelphia, PA 19104, USA
Mol Ther 14:328-35. 2006..AAV2/1 promises to be useful in further studies evaluating differentiation pathways of cochlear cells in health and disease and for developing gene-based therapies for congenital and acquired forms of peripheral hearing loss... - Gene therapy for ocular neovascularization: a cure in sightSamuel J Reich
FM Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, The University of Pennsylvania, 310 Stellar Chance Labs, 422 Curie Blvd, Philadelphia, Pennsylvania 19104 6069, USA
Curr Opin Genet Dev 13:317-22. 2003..A gene-therapy solution to the debilitating blindness caused by ocular neovascularization may be on the horizon... - Gene therapy for Leber congenital amaurosisJean Bennett
FM Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, 310 Stellar-Chance Labs, 422 Curie Boulevard, Philadelphia, PA 19104-2689, USA
Novartis Found Symp 255:195-202; discussion 202-7. 2004..Preliminary results of studies in progress are described and the implications of these results with respect to developing human clinical trials for LCA and for other retinal degenerative diseases are discussed... - Long-term protection of retinal structure but not function using RAAV.CNTF in animal models of retinitis pigmentosaF Q Liang
F. M. Kirby Center for Molecular Ophthalmology, Department of Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA 19104, USA
Mol Ther 4:461-72. 2001..GFP. The discordance of functional and structural results, especially in the rat models, points to the need for a greater understanding of the mechanism of action of CNTF before human application can be considered... - Gene delivery to the retina: from mouse to manJean Bennett
F M Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, Pennsylvania, USA
Methods Enzymol 507:255-74. 2012..The information presented here is designed to assist scientists and clinicians to use gene transfer to probe the biology of the retina and/or to move appropriate gene-based treatment studies from the bench to the clinic... - Gene therapy for Leber congenital amaurosisNadine S Dejneka
University of Pennsylvania, Scheie Eye Institute, Philadelphia, PA 19104-6069, USA
Adv Exp Med Biol 533:415-22. 2003 - Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkeyLuk H Vandenberghe
Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, and Center for Cellelar and Molecular Therapeutics, Children s Hospital of Philadelphia, Philadelphia, PA 19104, USA
Sci Transl Med 3:88ra54. 2011..These preclinical results provide guidance for optimal vector and dose selection in future human gene therapy trials to treat retinal diseases caused by loss of photoreceptors... - A deviant immune response to viral proteins and transgene product is generated on subretinal administration of adenovirus and adeno-associated virusVibha Anand
Univeristy of Pennsylvania, F. M. Kirby Center for Molecular Ophthalmology, 310 Stellar Chance Labs, Scheie Eye Institute, 422 Curie Blvd, Philadelphia, Pennsylvania 19104-6069, USA
Mol Ther 5:125-32. 2002..This subsequently generates a population of immunosuppressive Th2-type, cytokine-secreting, splenic T cells. This response may be advantageous to the development of ocular gene therapy... - Tie2Cre-mediated inactivation of plexinD1 results in congenital heart, vascular and skeletal defectsYing Zhang
Department of Cell and Developmental Biology, Cardiovascular Institute, University of Pennsylvania, Philadelphia, PA 19104, USA
Dev Biol 325:82-93. 2009..These results demonstrate an important role for PlexinD1 in embryonic and adult vasculature... - Imaging oxygen pressure in the rodent retina by phosphorescence lifetimeDavid F Wilson
Department of Biochemistry, Medical School, University of Pennsylvania, Philadelphia 19104, USA
Adv Exp Med Biol 578:119-24. 2006 - Pigment epithelium-derived factor expression in the developing mouse eyeK C Behling
F M Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia 19104 6069, USA
Mol Vis 8:449-54. 2002..Because of the potential importance of normal PEDF expression patterns in controlling ocular blood vessel growth in health and disease, we characterized these patterns over the period of retinal vascular development in the mouse... - Macular pigment and lutein supplementation in choroideremiaJacque L Duncan
Department of Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA 19104, USA
Exp Eye Res 74:371-81. 2002..There was no short-term change in the central vision of the patients on the supplement, but long-term influences of lutein supplementation on disease natural history warrant further study... - Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a modelA Auricchio
Institute for Human Gene Therapy, Department of Molecular and Cellular Engineering, The Wistar Institute, Philadelphia, PA, USA
Hum Mol Genet 10:3075-81. 2001..We conclude that pseudotyping provides a useful means to manipulate viral vector cell targeting specificity as well as retinal transduction characteristics of vectors containing the same genome... - Efficient trans-splicing in the retina expands the utility of adeno-associated virus as a vector for gene therapyS J Reich
F.M. Kirby Center for Molecular Ophthalmology, Department of Ophthalmology, Scheie Eye Institute, Philadelphia, PA 19104, USA
Hum Gene Ther 14:37-44. 2003..The use of AAV2/5 also increases the efficiency of trans-splicing in photoreceptors. Selection of the appropriate AAV serotype is likely to affect efficiency of trans-splicing in other organ systems as well... - Herpes simplex virus type 1 glycoprotein e is required for axonal localization of capsid, tegument, and membrane glycoproteinsFushan Wang
Department of Medicine, Division of Infectious Diseases, 502 Johnson Pavilion, University of Pennsylvania, Philadelphia, PA 19104-6073, USA
J Virol 79:13362-72. 2005..The requirement for gE in axonal targeting and retrograde spread highlights intriguing similarities and differences between HSV-1 and pseudorabies virus gE... - Oxygen distribution and vascular injury in the mouse eye measured by phosphorescence-lifetime imagingDavid F Wilson
Department of Biochemistry, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA
Appl Opt 44:5239-48. 2005..High-resolution maps of oxygen distribution in the retina can provide greater understanding of the role of oxygen and vascular function in diseases of the eye... - Pharmacological regulation of protein expression from adeno-associated viral vectors in the eyeAlberto Auricchio
Institute for Human Gene Therapy, The Wistar Institute, Department of Molecular and Cellular Engineering, University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA
Mol Ther 6:238-42. 2002..The rapamycin inducible system promises to be useful for developing gene therapies for inherited retinal degeneration and ocular neovascularization... - Retinal ganglion cell loss induced by acute optic neuritis in a relapsing model of multiple sclerosisKenneth S Shindler
Department of Ophthalmology, University of Pennsylvania, Scheie Eye Institute, 51 N 39th Street, Philadelphia, PA 19104, USA
Mult Scler 12:526-32. 2006..8% by day 18. No RGC loss occurred in eyes without optic neuritis. The fact that inflammation precedes RGC loss suggests that neuronal loss during optic neuritis occurs secondary to the inflammatory process... - Retinal progenitor cells--timing is everythingJean Bennett
Scheie Eye Institute, University of Pennsylvania, Philadelphia, USA
N Engl J Med 356:1577-9. 2007 - Gene therapy and animal models for retinal diseaseNadine S Dejneka
F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA
Dev Ophthalmol 37:188-98. 2003..We will describe the animal models used to study these disorders and emphasize the progress that has been made in using gene therapy for the treatment of retinal disease... - Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapyJ Bennett
Department of Ophthalmology, F M Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia 19104, USA
Nat Med 2:649-54. 1996..The findings demonstrate cell rescue by in vivo gene transfer, thus supporting the feasibility of treating an inherited retinal degeneration by somatic gene therapy... - Pharmacologically regulated gene expression in the retina following transduction with viral vectorsN S Dejneka
Department of Ophthalmology, Scheie Eye Institute, Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA 19104-6069, USA
Gene Ther 8:442-6. 2001..This mode of delivery and regulation offers a unique way to evaluate gene function in the eye and represents a novel method for introducing therapeutic proteins into the retina... - Disease expression of RP1 mutations causing autosomal dominant retinitis pigmentosaS G Jacobson
Department of Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, USA
Invest Ophthalmol Vis Sci 41:1898-908. 2000..To determine the disease expression in heterozygotes for mutations in the RP1 gene, a newly identified cause of autosomal dominant retinitis pigmentosa (adRP)... - Localization of pigment epithelium derived factor (PEDF) in developing and adult human ocular tissuesP C Karakousis
F M Kirby Center for Molecular Ophthalmology, University of Pennsylvania, Philadelphia, PA 19104, USA
Mol Vis 7:154-63. 2001..To localize pigment epithelium-derived factor (PEDF) in developing and adult human ocular tissues... - Dissecting the axoneme interactome: the mammalian orthologue of Chlamydomonas PF6 interacts with sperm-associated antigen 6, the mammalian orthologue of Chlamydomonas PF16Zhibing Zhang
Center for Research on Reproduction and Women s Health and the Department of Ophthalmology, University of Pennsylvania Medical Center, Philadelphia, Pennsylvania 19104, USA
Mol Cell Proteomics 4:914-23. 2005..Thus, PF6, SPAG6, and PF20 form a newly identified network that links together components of the axoneme central apparatus and presumably participates in its dynamic regulation of ciliary and flagellar beat... - Immune response following intraocular delivery of recombinant viral vectorsJ Bennett
FM Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA 19104-6069, USA
Gene Ther 10:977-82. 2003..Neither AAV nor lentivirus, however, elicit a cell-mediated response and are thus promising vectors for treatment of chronic ocular (retinal) diseases... - The bionic retina: a small molecule with big potential for visual restorationTheodore G Drivas
F M Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA 19104, USA
Neuron 75:185-7. 2012..Results show that, with the appropriate illumination, AAQ restores light sensitivity and visual behavior... - Increased expression of ceruloplasmin in the retina following photic injuryLin Chen
F M Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA
Mol Vis 9:151-8. 2003..The purpose of this study was to determine whether retinal ceruloplasmin is upregulated following photo-oxidation, to localize ceruloplasmin protein, and to determine which ceruloplasmin isoform is present in the retina... - Novel mutation in the TIMP3 gene causes Sorsby fundus dystrophySamuel G Jacobson
Scheie Eye Institute, 51 N 39th St, Philadelphia, PA 19104, USA
Arch Ophthalmol 120:376-9. 2002..To determine the molecular basis of a retinopathy previously described as dominant macular subretinal neovascularization with peripheral retinal degeneration... - Adenovirus-mediated delivery of catalase to retinal pigment epithelial cells protects neighboring photoreceptors from photo-oxidative stressT S Rex
F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA 19104, USA
Hum Gene Ther 15:960-7. 2004..The data provide a paradigm for antioxidant gene therapy with catalase, designed to protect not only transduced cells, but also neighboring cells... - Gene therapy and retinitis pigmentosa: advances and future challengesN S Dejneka
F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia 19104-6069, USA
Bioessays 23:662-8. 2001..Future challenges include confirming therapeutic effects in animal models with eyes more anatomically similar to those of humans and demonstrating long-term rescue with minimal toxicity... - Adeno-Associated Virus-Mediated Gene Transfer to Renal Tubule Cells via a Retrograde Ureteral ApproachDaniel C Chung
Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, USA
Nephron Extra 1:217-23. 2011..Gene therapy involves delivery of exogenous DNA to provide a therapeutic protein. Ideally, a gene therapy vector should be non-toxic, non-immunogenic, easy to produce, and efficient in protecting and delivering DNA into target cells... - Spectrum of mutations in the RPGR gene that are identified in 20% of families with X-linked retinitis pigmentosaM Buraczynska
Department of Ophthalmology, W K Kellogg Eye Center, University of Michigan, Ann Arbor 48105, USA
Am J Hum Genet 61:1287-92. 1997..The reported studies will be beneficial in establishing genotype-phenotype correlations and should lead to further investigations seeking to understand the mechanism of disease pathogenesis... - Melatonin delays photoreceptor degeneration in the rds/rds mouseF Q Liang
Department of Ophthalmology, Scheie Eye Institute, University of Pennsylvania School of Medicine, Philadelphia 19104, USA
Neuroreport 12:1011-4. 2001..Melatonin treatment significantly delayed photoreceptor loss and reduced the number of apoptotic photoreceptors. Further studies should determine if melatonin will have potential for the treatment of certain human retinal degenerations... - Strain-dependent anterior segment neovascularization following intravitreal gene transfer of basic fibroblast growth factor (bFGF)A R Gupta
Department of Ophthalmology, University of Pennsylvania, Philadelphia, USA
J Gene Med 3:252-9. 2001..Genetic modifiers may contribute to and/or prevent neovascularization induced by bFGF... - Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in miceMariacarmela Allocca
Telethon Institute of Genetics and Medicine TIGEM, Naples, Italy
J Clin Invest 118:1955-64. 2008..The possibility of packaging large genes in AAV greatly expands the therapeutic potential of this vector system... - Strategies for delivery of rod-derived cone viability factorJean Bennett
Ophthalmology, Cell and Developmental Biology at the University of Pennsylvania School of Medicine in Philadelphia, PA, USA
Retina 25:S47. 2005 - Lateral connectivity and contextual interactions in macaque primary visual cortexDan D Stettler
The Rockefeller University, 1230 York Avenue, New York, NY 10021, USA
Neuron 36:739-50. 2002..These findings suggest that V1 intrinsic horizontal connections provide a more likely substrate for contour integration... - Adeno-associated virus encoding green fluorescent protein as a label for retinal pigment epitheliumKathleen A Hansen
Institute of Ophthalmology and Visual Science, New Jersey Medical School, University of Medicine and Dentistry of New Jersey, Newark, New Jersey 01701, USA
Invest Ophthalmol Vis Sci 44:772-80. 2003..Cell division may cause dilution of the label, and release of cell contents into the subretinal space may cause label transfer to photoreceptors. Exposure to light of transduced cells should be limited... - Müller cell transduction by AAV2 in normal and degenerative retinasFong-Qi Liang
Retina Foundation of the Southwest, 9900 N. Central Expressway, Suite 400, Dallas, TX 75231, USA
Adv Exp Med Biol 533:439-45. 2003 - Imaging oxygen pressure in the retina of the mouse eyeDavid F Wilson
Adv Exp Med Biol 566:159-65. 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 blindnessGregory M Acland
Baker Institute, Cornell University, Hungerford Hill Road, Ithaca, NY 14853, USA
Mol Ther 12:1072-82. 2005..Successful, stable restoration of rod and cone photoreceptor function in these dogs has important implications for treatment of human patients affected with Leber congenital amaurosis caused by RPE65 mutations... - Eye movement recordings as an effectiveness indicator of gene therapy in RPE65-deficient canines: implications for the ocular motor systemJonathan B Jacobs
Daroff-Dell'Osso Ocular Motility Laboratory, Veterans Affairs Medical Center, Department of Neurology, Case Western Reserve University and University Hospital of Cleveland, OH 44106, USA
Invest Ophthalmol Vis Sci 47:2865-75. 2006..IN improvement, as quantified by the NAFX, is an objective motor indicator of visual improvement due to gene therapy... - Accelerated mortality from hydrocephalus and pneumonia in mice with a combined deficiency of SPAG6 and SPAG16L reveals a functional interrelationship between the two central apparatus proteinsZhibing Zhang
Department of Obstetrics and Gynecology, Virginia Commonwealth University, Richmond, Virginia 23298, USA
Cell Motil Cytoskeleton 64:360-76. 2007..The findings also demonstrate that SPAG6 and SPAG16L have related roles in controlling the function of cilia in the brain and lung...
Research Grants
- Animal Model for a Hereditary Macular DegenerationJean Bennett; Fiscal Year: 2004..These studies aim to reveal specific biological pathways relevant to the pathology in this animal model. Any such pathways could potentially be manipulated in the future to slow/prevent the disease process. ..
- Gene Therapy for Inherited Retinal DegenerationJean Bennett; Fiscal Year: 2007..The ability to rescue photoreceptors in the PDEbeta or abcr -based animal models could ultimately pave the way for genetic therapy as a treatment for blinding retinal diseases. ..
- AAV-mediated gene correction in retinaJean Bennett; Fiscal Year: 2007....
- Gene Therapy for Usher Syndrome (USH1C)Jean Bennett; Fiscal Year: 2007....
- GENETIC THERAPY FOR HEREDITARY RETINAL DEGENERATIONSJean Bennett; Fiscal Year: 2002..The ability to rescue photoreceptors in the betaPDE or rhodopsin-based animal models could ultimately pave the way for genetic therapy as a treatment for blinding and currently incurable inherited retinal degenerations such as RP. ..
- ANIMAL MODELS FOR A HEREDITARY MACULAR DEGENERATONJean Bennett; Fiscal Year: 2000..The characterization of the pathogenic basis of SFD could suggest therapeutic approaches for treating this disease and other forms of macular degeneration. ..