Repair of Retinal Degeneration following Ex Vivo Minicircle DNA Gene Therapy and Transplantation of Corrected Photoreceptor Progenitors.
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D. Fischer | R. MacLaren | Mandeep S. Singh | A. Barnard | S. D. De Silva | M. McClements | Alona Barnea-Cramer
[1] J. B. Demb,et al. Restoration of vision after de novo genesis of rod photoreceptors in mammalian retinas , 2018, Nature.
[2] Albrecht Rothermel,et al. Interim Results of a Multicenter Trial with the New Electronic Subretinal Implant Alpha AMS in 15 Patients Blind from Inherited Retinal Degenerations , 2017, Front. Neurosci..
[3] Takashi Daimon,et al. Autologous Induced Stem‐Cell–Derived Retinal Cells for Macular Degeneration: Brief Report , 2017, The New England journal of medicine.
[4] Genshiro A. Sunagawa,et al. iPSC-Derived Retina Transplants Improve Vision in rd1 End-Stage Retinal-Degeneration Mice , 2017, Stem cell reports.
[5] R. MacLaren,et al. Transplanted photoreceptor precursors transfer proteins to host photoreceptors by a mechanism of cytoplasmic fusion , 2016, Nature Communications.
[6] Oliver Borsch,et al. Retinal transplantation of photoreceptors results in donor–host cytoplasmic exchange , 2016, Nature Communications.
[7] E. L. West,et al. Donor and host photoreceptors engage in material transfer following transplantation of post-mitotic photoreceptor precursors , 2016, Nature Communications.
[8] R. MacLaren,et al. Function of human pluripotent stem cell-derived photoreceptor progenitors in blind mice , 2016, Scientific Reports.
[9] K. Gregory-Evans,et al. An ex vivo gene therapy approach in X-linked retinoschisis , 2016, Molecular vision.
[10] P. Carmeliet,et al. Stem Cell-Derived Photoreceptor Transplants Differentially Integrate Into Mouse Models of Cone-Rod Dystrophy. , 2016, Investigative ophthalmology & visual science.
[11] P. Charbel Issa,et al. Single residue AAV capsid mutation improves transduction of photoreceptors in the Abca4-/- mouse and bipolar cells in the rd1 mouse and human retina ex-vivo , 2016, Gene Therapy.
[12] A. Bassuk,et al. Precision Medicine: Genetic Repair of Retinitis Pigmentosa in Patient-Derived Stem Cells , 2016, Scientific Reports.
[13] R. MacLaren,et al. CNTF Gene Therapy Confers Lifelong Neuroprotection in a Mouse Model of Human Retinitis Pigmentosa. , 2015, Molecular therapy : the journal of the American Society of Gene Therapy.
[14] Fani Sousa,et al. Minicircle DNA vectors for gene therapy: advances and applications , 2015, Expert opinion on biological therapy.
[15] U. Wolfrum,et al. Translational read-through of the RP2 Arg120stop mutation in patient iPSC-derived retinal pigment epithelium cells , 2014, Human molecular genetics.
[16] S. Tsang,et al. Gene therapy in patient-specific stem cell lines and a preclinical model of retinitis pigmentosa with membrane frizzled-related protein defects. , 2014, Molecular therapy : the journal of the American Society of Gene Therapy.
[17] H. Okano,et al. The use of induced pluripotent stem cells to reveal pathogenic gene mutations and explore treatments for retinitis pigmentosa , 2014, Molecular Brain.
[18] E. Stone,et al. CEP290 gene transfer rescues Leber congenital amaurosis cellular phenotype , 2014, Gene Therapy.
[19] R. MacLaren,et al. Cell fusion following photoreceptor transplantation into the non-degenerate retina , 2014 .
[20] E. Stone,et al. Photoreceptor cells with profound structural deficits can support useful vision in mice. , 2014, Investigative ophthalmology & visual science.
[21] M. Ader,et al. Subretinal transplantation of MACS purified photoreceptor precursor cells into the adult mouse retina. , 2014, Journal of visualized experiments : JoVE.
[22] Angelika Braun,et al. Artificial vision with wirelessly powered subretinal electronic implant alpha-IMS , 2013, Proceedings of the Royal Society B: Biological Sciences.
[23] R. MacLaren,et al. Translating induced pluripotent stem cells from bench to bedside: application to retinal diseases. , 2013, Current gene therapy.
[24] R. MacLaren,et al. Clinical applications of retinal gene therapy , 2013, Progress in Retinal and Eye Research.
[25] Peter Charbel Issa,et al. Reversal of end-stage retinal degeneration and restoration of visual function by photoreceptor transplantation , 2013, Proceedings of the National Academy of Sciences.
[26] E. L. West,et al. Repair of the degenerate retina by photoreceptor transplantation , 2012, Proceedings of the National Academy of Sciences.
[27] E. L. West,et al. Defining the Integration Capacity of Embryonic Stem Cell‐Derived Photoreceptor Precursors , 2012, Stem cells.
[28] E. L. West,et al. Restoration of vision after transplantation of photoreceptors , 2012, Nature.
[29] F. Delori,et al. Optimization of in vivo confocal autofluorescence imaging of the ocular fundus in mice and its application to models of human retinal degeneration. , 2012, Investigative ophthalmology & visual science.
[30] D. Corbeil,et al. Increased integration of transplanted CD73-positive photoreceptor precursors into adult mouse retina. , 2011, Investigative ophthalmology & visual science.
[31] K. Gregory-Evans,et al. Pharmacological Enhancement of ex vivo Gene Therapy Neuroprotection in a Rodent Model of Retinal Degeneration , 2011, Ophthalmic Research.
[32] G. Freeman,et al. © The American Society of Gene & Cell Therapy original article Minicircle DNA-based Gene Therapy Coupled With Immune Modulation Permits Long-term Expression of α-l-Iduronidase in Mice With , 2022 .
[33] V. Sheffield,et al. Light aversion in mice depends on nonimage-forming irradiance detection. , 2010, Behavioral neuroscience.
[34] Alfred Stett,et al. Subretinal electronic chips allow blind patients to read letters and combine them to words , 2010, Proceedings of the Royal Society B: Biological Sciences.
[35] Shannon M. Conley,et al. Nanoparticles for retinal gene therapy , 2010, Progress in Retinal and Eye Research.
[36] G. Daley,et al. Transplantation of Adult Mouse iPS Cell-Derived Photoreceptor Precursors Restores Retinal Structure and Function in Degenerative Mice , 2010, PloS one.
[37] Shannon M. Conley,et al. Gene delivery to mitotic and postmitotic photoreceptors Via compacted DNA nanoparticles results in improved phenotype in a mouse model of retinitis pigmentosa , 2010, FASEB journal : official publication of the Federation of American Societies for Experimental Biology.
[38] Elliot M. Frohman,et al. Direct and consensual murine pupillary reflex metrics: Establishing normative values , 2009, Autonomic Neuroscience.
[39] K. Gregory-Evans,et al. Ex vivo gene therapy using intravitreal injection of GDNF-secreting mouse embryonic stem cells in a rat model of retinal degeneration , 2009, Molecular vision.
[40] Shannon M. Conley,et al. A Partial Structural and Functional Rescue of a Retinitis Pigmentosa Model with Compacted DNA Nanoparticles , 2009, PloS one.
[41] T. Reh,et al. Transplantation of human embryonic stem cell-derived photoreceptors restores some visual function in Crx-deficient mice. , 2009, Cell stem cell.
[42] M. Kay,et al. Gene Transfer to Mouse Heart and Skeletal Muscles Using a Minicircle Expressing Human Vascular Endothelial Growth Factor , 2009, Journal of cardiovascular pharmacology.
[43] M. Kay,et al. Novel Minicircle Vector for Gene Therapy in Murine Myocardial Infarction , 2009, Circulation.
[44] K. Martin,et al. Development and characterization of an adult retinal explant organotypic tissue culture system as an in vitro intraocular stem cell transplantation model. , 2008, Investigative ophthalmology & visual science.
[45] M. O'Reilly,et al. A transgenic mouse model for gene therapy of rhodopsin-linked Retinitis Pigmentosa , 2008, Vision Research.
[46] S. W. Kim,et al. Efficient expression of vascular endothelial growth factor using minicircle DNA for angiogenic gene therapy. , 2008, Journal of controlled release : official journal of the Controlled Release Society.
[47] W. Jechlinger. Optimization and delivery of plasmid DNA for vaccination , 2006, Expert review of vaccines.
[48] T. Miyakawa,et al. Light/dark Transition Test for Mice , 2006, Journal of visualized experiments : JoVE.
[49] T. Salt,et al. Retinal repair by transplantation of photoreceptor precursors , 2006, Nature.
[50] S. D. De Smedt,et al. Influence of plasmid DNA topology on the transfection properties of DOTAP/DOPE lipoplexes. , 2006, Journal of controlled release : official journal of the Controlled Release Society.
[51] Yixin Zeng,et al. Minicircle-IFNγ Induces Antiproliferative and Antitumoral Effects in Human Nasopharyngeal Carcinoma , 2006, Clinical Cancer Research.
[52] Qiliang Li,et al. Investigations of the effect of DNA size in transient transfection assay using dual luciferase system. , 2005, Analytical biochemistry.
[53] R M Douglas,et al. Independent visual threshold measurements in the two eyes of freely moving rats and mice using a virtual-reality optokinetic system , 2005, Visual Neuroscience.
[54] M. Kay,et al. Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo , 2004, Gene Therapy.
[55] Krzysztof Palczewski,et al. Impairment of the transient pupillary light reflex in Rpe65(-/-) mice and humans with leber congenital amaurosis. , 2004, Investigative ophthalmology & visual science.
[56] M. Kay,et al. Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo. , 2003, Molecular therapy : the journal of the American Society of Gene Therapy.
[57] M. Hascöet,et al. The mouse light/dark box test. , 2003, European journal of pharmacology.
[58] I. Jackson,et al. Presence of visual head tracking differentiates normal sighted from retinal degenerate mice , 2002, Neuroscience Letters.
[59] M. Kay,et al. Linear DNAs concatemerize in vivo and result in sustained transgene expression in mouse liver. , 2001, Molecular therapy : the journal of the American Society of Gene Therapy.
[60] M. Seeliger,et al. Evaluation of the rhodopsin knockout mouse as a model of pure cone function. , 2001, Investigative ophthalmology & visual science.
[61] D. Scherman,et al. Plasmid DNA size does not affect the physicochemical properties of lipoplexes but modulates gene transfer efficiency. , 1999, Nucleic acids research.
[62] D. Scherman,et al. Minicircle: an improved DNA molecule for in vitro and in vivo gene transfer , 1999, Gene Therapy.
[63] R L Sidman,et al. Morphological, physiological, and biochemical changes in rhodopsin knockout mice. , 1999, Proceedings of the National Academy of Sciences of the United States of America.
[64] P Strata,et al. The Early Phase of Horizontal Optokinetic Responses in the Pigmented Rat and the Effects of Lesions of the Visual Cortex , 1997, Vision Research.
[65] P. Sieving,et al. Retinopathy induced in mice by targeted disruption of the rhodopsin gene , 1997, Nature Genetics.
[66] B Rosner,et al. The relationship between visual field size and electroretinogram amplitude in retinitis pigmentosa. , 1996, Investigative ophthalmology & visual science.
[67] M. Naash,et al. Properties of the mouse cone-mediated electroretinogram during light adaptation , 1993, Neuroscience Letters.
[68] R. Lund,et al. Development of Light‐activated Pupilloconstriction in Rats as Mediated by Normal and Transplanted Retinae , 1992, The European journal of neuroscience.
[69] T. L. McGee,et al. Mutations within the rhodopsin gene in patients with autosomal dominant retinitis pigmentosa. , 1990, The New England journal of medicine.
[70] David W. Yandell,et al. A point mutation of the rhodopsin gene in one form of retinitis pigmentosa , 1990, Nature.
[71] C. Belzung,et al. Behavioural Validation of a Light/Dark Choice Procedure for Testing Anti-Anxiety Agents , 1989, Behavioural Processes.
[72] F. Goodwin,et al. Preliminary report of a simple animal behavior model for the anxiolytic effects of benzodiazepines , 1980, Pharmacology Biochemistry and Behavior.
[73] Wenzhong Li,et al. Non-viral gene delivery methods. , 2013, Current pharmaceutical biotechnology.
[74] A. Fire,et al. © The American Society of Gene & Cell Therapy original article Minicircle DNA Vectors Achieve Sustained Expression Reflected by Active Chromatin and Transcriptional , 2022 .
[75] P. Schlag,et al. Performance of High Quality Minicircle DNA for In Vitro and In Vivo Gene Transfer , 2012, Molecular Biotechnology.
[76] R. MacLaren,et al. Non‐viral retinal gene therapy: a review , 2012, Clinical & experimental ophthalmology.
[77] Peter Mayrhofer,et al. Use of minicircle plasmids for gene therapy. , 2009, Methods in molecular biology.
[78] U. Grüsser-Cornehls,et al. Horizontal optokinetic ocular nystagmus in wildtype (B6CBA+/+) and weaver mutant mice , 2004, Experimental Brain Research.