| scholarly article | Q13442814 |
| review article | Q7318358 |
| P356 | DOI | 10.1089/HUM.2015.030 |
| P8608 | Fatcat ID | release_4hduuv5irnh6hh667gzyb6mol4 |
| P3181 | OpenCitations bibliographic resource ID | 1651589 |
| P932 | PMC publication ID | 4410187 |
| P698 | PubMed publication ID | 25762209 |
| P50 | author | Enrico M. Surace | Q47504013 |
| Sandro Banfi | Q56581073 | ||
| Alberto Auricchio | Q56950196 | ||
| Ivana Trapani | Q57306425 | ||
| Francesca Simonelli | Q37384848 | ||
| P2093 | author name string | Alberto Auricchio | |
| Francesca Simonelli | |||
| Ivana Trapani | |||
| Enrico M. Surace | |||
| P2860 | cites work | Expression of human factor VIII by splicing between dimerized AAV vectors | Q45886212 |
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| Effect of genome size on AAV vector packaging | Q33730405 | ||
| Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome > or = 8.2 kb. | Q33730469 | ||
| Gene delivery to mitotic and postmitotic photoreceptors via compacted DNA nanoparticles results in improved phenotype in a mouse model of retinitis pigmentosa | Q33752902 | ||
| Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success | Q33771032 | ||
| Molecular genetics of human retinal disease | Q33847668 | ||
| Gene therapy for blindness | Q34037010 | ||
| Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa | Q34158497 | ||
| Engineering synthetic TALE and CRISPR/Cas9 transcription factors for regulating gene expression | Q34243548 | ||
| Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6. | Q34473295 | ||
| Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina | Q34556855 | ||
| Vector platforms for gene therapy of inherited retinopathies. | Q34561531 | ||
| Lentiviral gene replacement therapy of retinas in a mouse model for Usher syndrome type 1B. | Q34608354 | ||
| Safety and effects of the vector for the Leber hereditary optic neuropathy gene therapy clinical trial. | Q34700126 | ||
| Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors | Q34700925 | ||
| Pseudo-fovea formation after gene therapy for RPE65-LCA. | Q34999841 | ||
| Clinical characteristics and current therapies for inherited retinal degenerations | Q35045067 | ||
| EIAV-based retinal gene therapy in the shaker1 mouse model for usher syndrome type 1B: development of UshStat | Q35140281 | ||
| Ribozyme rescue of photoreceptor cells in P23H transgenic rats: long-term survival and late-stage therapy. | Q35306158 | ||
| Preservation of photoreceptor morphology and function in P23H rats using an allele independent ribozyme. | Q35603911 | ||
| Novel mini-dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma | Q35678425 | ||
| Adenovirus-associated virus vector-mediated gene transfer in hemophilia B | Q35691659 | ||
| Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy | Q35799349 | ||
| Nanoparticle-based technologies for retinal gene therapy | Q35843512 | ||
| DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice. | Q36190772 | ||
| Surfaces of rod photoreceptor disk membranes: light-activated enzymes | Q36209402 | ||
| Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice. | Q36545533 | ||
| Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector | Q36586077 | ||
| Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement | Q36598421 | ||
| Determining consequences of retinal membrane guanylyl cyclase (RetGC1) deficiency in human Leber congenital amaurosis en route to therapy: residual cone-photoreceptor vision correlates with biochemical properties of the mutants | Q36709692 | ||
| Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus | Q36808032 | ||
| Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2 | Q36906324 | ||
| Transduction of photoreceptors with equine infectious anemia virus lentiviral vectors: safety and biodistribution of StarGen for Stargardt disease | Q36926064 | ||
| Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors | Q36968876 | ||
| Dual AAV therapy ameliorates exercise-induced muscle injury and functional ischemia in murine models of Duchenne muscular dystrophy | Q37111962 | ||
| Barriers for retinal gene therapy: separating fact from fiction | Q37195446 | ||
| Functional rescue of degenerating photoreceptors in mice homozygous for a hypomorphic cGMP phosphodiesterase 6 b allele (Pde6bH620Q). | Q37273993 | ||
| The role of the photoreceptor ABC transporter ABCA4 in lipid transport and Stargardt macular degeneration | Q37351985 | ||
| Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa. | Q37376742 | ||
| Oversized AAV transductifon is mediated via a DNA-PKcs-independent, Rad51C-dependent repair pathway | Q37393799 | ||
| Intravitreal delivery of AAV8 retinoschisin results in cell type-specific gene expression and retinal rescue in the Rs1-KO mouse | Q37414094 | ||
| Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach | Q37432588 | ||
| Effective delivery of large genes to the retina by dual AAV vectors | Q37591108 | ||
| Leber congenital amaurosis: clinical correlations with genotypes, gene therapy trials update, and future directions | Q37652856 | ||
| Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A. | Q37710671 | ||
| The molecular basis of human retinal and vitreoretinal diseases | Q37723484 | ||
| Clinical applications of retinal gene therapy. | Q38045154 | ||
| Gene therapy for inherited retinal degenerations | Q38202230 | ||
| Gene therapy for mitochondrial diseases: Leber Hereditary Optic Neuropathy as the first candidate for a clinical trial | Q38202231 | ||
| Development of photoreceptor-specific promoters and their utility to investigate EIAV lentiviral vector mediated gene transfer to photoreceptors | Q38297007 | ||
| Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy | Q39505058 | ||
| AAV-mediated gene delivery in Dp71-null mouse model with compromised barriers | Q39545173 | ||
| Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer | Q39551137 | ||
| Little vector, big gene transduction: fragmented genome reassembly of adeno-associated virus | Q39639471 | ||
| The relationship of primate foveal cones to the pigment epithelium | Q39666483 | ||
| Clinical and molecular genetics of Leber's congenital amaurosis: a multicenter study of Italian patients | Q40179772 | ||
| Gene therapy in the second eye of RPE65-deficient dogs improves retinal function | Q41835416 | ||
| Long-term rescue of retinal structure and function by rhodopsin RNA replacement with a single adeno-associated viral vector in P23H RHO transgenic mice. | Q42003725 | ||
| Retinal transduction profiles by high-capacity viral vectors | Q42208596 | ||
| Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors | Q42235082 | ||
| RNA interference-mediated suppression and replacement of human rhodopsin in vivo | Q42413344 | ||
| Suppression of mouse rhodopsin expression in vivo by AAV mediated siRNA delivery | Q42746387 | ||
| Changes in adeno-associated virus-mediated gene delivery in retinal degeneration | Q42840509 | ||
| Lentiviral vector-mediated gene transfer in adult mouse photoreceptors is impaired by the presence of a physical barrier | Q43564494 | ||
| Gene therapy restores vision in a canine model of childhood blindness | Q43590733 | ||
| Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model | Q43829645 | ||
| Retinal cell type expression specificity of HIV-1-derived gene transfer vectors upon subretinal injection in the adult rat: influence of pseudotyping and promoter. | Q43833846 | ||
| In vivo gene transfer to the mouse eye using an HIV-based lentiviral vector; efficient long-term transduction of corneal endothelium and retinal pigment epithelium | Q43917989 | ||
| Efficient trans-splicing in the retina expands the utility of adeno-associated virus as a vector for gene therapy. | Q44305893 | ||
| Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog. | Q44376450 | ||
| Adenoviral-mediated gene transfer to retinal explants during development and degeneration | Q44706879 | ||
| In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous | Q45350780 | ||
| Adeno-associated virus mediated gene therapy for retinal degenerative diseases. | Q45362093 | ||
| Efficiency of lentiviral transduction during development in normal and rd mice | Q45861263 | ||
| Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium | Q45863029 | ||
| Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a quantitative comparison | Q45880308 | ||
| P433 | issue | 4 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | gene therapy | Q213901 |
| P304 | page(s) | 193-200 | |
| P577 | publication date | 2015-04-01 | |
| P1433 | published in | Human Gene Therapy | Q15757580 |
| P1476 | title | Gene therapy of inherited retinal degenerations: prospects and challenges | |
| P478 | volume | 26 |
| Q37190609 | Advances in Gene Therapy for Diseases of the Eye. |
| Q90656139 | Attenuation of Inherited and Acquired Retinal Degeneration Progression with Gene-based Techniques |
| Q54390675 | Cell metabolism: Sugar for sight. |
| Q45875203 | Dual AAV Vectors for Stargardt Disease |
| Q33729610 | Harnessing the Potential of Human Pluripotent Stem Cells and Gene Editing for the Treatment of Retinal Degeneration |
| Q42380127 | How Azobenzene Photoswitches Restore Visual Responses to the Blind Retina |
| Q37631687 | Neuroprotective Strategy in Retinal Degeneration: Suppressing ER Stress-Induced Cell Death via Inhibition of the mTOR Signal |
| Q52684281 | New technologies for developing second generation retinal prostheses. |
| Q47364210 | Phenotypic characterization of P23H and S334ter rhodopsin transgenic rat models of inherited retinal degeneration |
| Q47554759 | Recent Advancements in Gene Therapy for Hereditary Retinal Dystrophies |
| Q37452246 | Reprogramming metabolism by targeting sirtuin 6 attenuates retinal degeneration |
| Q37481542 | Significant changes in endogenous retinal gene expression assessed 1 year after a single intraocular injection of AAV-CNTF or AAV-BDNF |
| Q88781061 | The Eyes Have it: A Rheumatologist's View of Uveitis |
| Q47769050 | Toward smart design of retinal drug carriers: a novel bovine retinal explant model to study the barrier role of the vitreoretinal interface |
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