| scholarly article | Q13442814 |
| P819 | ADS bibcode | 2014PLoSO...992928B |
| P356 | DOI | 10.1371/JOURNAL.PONE.0092928 |
| P8608 | Fatcat ID | release_g6aed653dzbd7b7vdska6e4om4 |
| P932 | PMC publication ID | 3966841 |
| P698 | PubMed publication ID | 24671090 |
| P5875 | ResearchGate publication ID | 261138413 |
| P50 | author | Anand Swaroop | Q30506132 |
| Samuel G. Jacobson | Q42410256 | ||
| William W Hauswirth | Q64478429 | ||
| Wei-Chieh Huang | Q88565352 | ||
| Artur V Cideciyan | Q90596707 | ||
| Sanford L Boye | Q92324390 | ||
| Alexander Sumaroka | Q116997512 | ||
| Edwin M. Stone | Q37381855 | ||
| Budd A. Tucker | Q38326878 | ||
| P2093 | author name string | Alejandro J Roman | |
| Qing Ruan | |||
| Melani B Olivares | |||
| Shannon E Boye | |||
| Renee C Ryals | |||
| P2860 | cites work | Gene therapy rescues cone function in congenital achromatopsia | Q24595787 |
| Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial | Q24608049 | ||
| Disruption of CEP290 microtubule/membrane-binding domains causes retinal degeneration | Q24617850 | ||
| Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy | Q24621941 | ||
| Safety and efficacy of gene transfer for Leber's congenital amaurosis | Q24634724 | ||
| In-frame deletion in a novel centrosomal/ciliary protein CEP290/NPHP6 perturbs its interaction with RPGR and results in early-onset retinal degeneration in the rd16 mouse | Q24671808 | ||
| A comprehensive review of retinal gene therapy | Q27023914 | ||
| Genetic and physical interaction between the NPHP5 and NPHP6 gene products | Q28118065 | ||
| Effect of gene therapy on visual function in Leber's congenital amaurosis | Q28277981 | ||
| Centrosomal-ciliary gene CEP290/NPHP6 mutations result in blindness with unexpected sparing of photoreceptors and visual brain: implications for therapy of Leber congenital amaurosis | Q28305398 | ||
| CRB1 is essential for external limiting membrane integrity and photoreceptor morphogenesis in the mammalian retina | Q28508050 | ||
| Restoration of cone vision in a mouse model of achromatopsia | Q28509747 | ||
| Nrl is required for rod photoreceptor development | Q28595099 | ||
| Cone photoreceptors are the main targets for gene therapy of NPHP5 (IQCB1) or NPHP6 (CEP290) blindness: generation of an all-cone Nphp6 hypomorph mouse that mimics the human retinal ciliopathy | Q30498337 | ||
| The N-terminal region of centrosomal protein 290 (CEP290) restores vision in a zebrafish model of human blindness. | Q30499095 | ||
| The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina. | Q30525669 | ||
| Myosin7a deficiency results in reduced retinal activity which is improved by gene therapy | Q31132177 | ||
| In vivo three-dimensional high-resolution imaging of rodent retina with spectral-domain optical coherence tomography | Q33280191 | ||
| Characterization of genome integrity for oversized recombinant AAV vector | Q33576852 | ||
| From the laboratory to the clinic: molecular genetic testing in pediatric ophthalmology | Q33617837 | ||
| 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 | ||
| Electroretinographic analyses of Rpe65-mutant rd12 mice: developing an in vivo bioassay for human gene therapy trials of Leber congenital amaurosis. | Q40160729 | ||
| Trans-splicing vectors expand the packaging limits of adeno-associated virus for gene therapy applications. | Q40677897 | ||
| Recombinant AAV-mediated gene delivery using dual vector heterodimerization. | Q40746276 | ||
| 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 | ||
| Efficiency of lentiviral transduction during development in normal and rd mice | Q45861263 | ||
| AAV-mediated expression targeting of rod and cone photoreceptors with a human rhodopsin kinase promoter | Q45870501 | ||
| Design of trans-splicing adeno-associated viral vectors for Duchenne muscular dystrophy gene therapy | Q45879448 | ||
| Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a quantitative comparison | Q45880308 | ||
| Retinal optogenetic therapies: clinical criteria for candidacy | Q45886746 | ||
| Stable and efficient intraocular gene transfer using pseudotyped EIAV lentiviral vectors | Q45888728 | ||
| Gene delivery to the retina using lentiviral vectors | Q46876210 | ||
| RPE65 is essential for the function of cone photoreceptors in NRL-deficient mice | Q47035993 | ||
| Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene. | Q51712353 | ||
| Efficient recovery of dysferlin deficiency by dual adeno-associated vector-mediated gene transfer | Q82892069 | ||
| Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success | Q33771032 | ||
| Differential macular morphology in patients with RPE65-, CEP290-, GUCY2D-, and AIPL1-related Leber congenital amaurosis | Q33845544 | ||
| Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy | Q33992484 | ||
| Leber congenital amaurosis: genes, proteins and disease mechanisms | Q34796759 | ||
| Protective effects of resveratrol in experimental retinal detachment | Q34990639 | ||
| Gene therapy for red-green colour blindness in adult primates | Q35003425 | ||
| In vivo function of the orphan nuclear receptor NR2E3 in establishing photoreceptor identity during mammalian retinal development | Q35070506 | ||
| Accumulation of the Raf-1 kinase inhibitory protein (Rkip) is associated with Cep290-mediated photoreceptor degeneration in ciliopathies | Q35150162 | ||
| Human CRB1-associated retinal degeneration: comparison with the rd8 Crb1-mutant mouse model | Q35221208 | ||
| Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse. | Q35518971 | ||
| Native molecular state of adeno-associated viral vectors revealed by single-molecule sequencing | Q35678415 | ||
| Novel mini-dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma | Q35678425 | ||
| Ocular and extra-ocular features of patients with Leber congenital amaurosis and mutations in CEP290. | Q35768517 | ||
| Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy | Q35799349 | ||
| Nrl-knockout mice deficient in Rpe65 fail to synthesize 11-cis retinal and cone outer segments | Q35861731 | ||
| Increased cone sensitivity to ABCA4 deficiency provides insight into macular vision loss in Stargardt's dystrophy | Q35957421 | ||
| Cellular remodeling in mammalian retina: results from studies of experimental retinal detachment | Q36040003 | ||
| RPGR-associated retinal degeneration in human X-linked RP and a murine model | Q36173078 | ||
| Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice. | Q36545533 | ||
| Photoreceptor sensory cilia and ciliopathies: focus on CEP290, RPGR and their interacting proteins | Q36585455 | ||
| Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector | Q36586077 | ||
| Preservation of cone photoreceptors after a rapid yet transient degeneration and remodeling in cone-only Nrl-/- mouse retina | Q36595691 | ||
| Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus | Q36808032 | ||
| Transduction of photoreceptors with equine infectious anemia virus lentiviral vectors: safety and biodistribution of StarGen for Stargardt disease | Q36926064 | ||
| Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics | Q36937189 | ||
| Visual acuity changes in patients with leber congenital amaurosis and mutations in CEP290 | Q36944797 | ||
| Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors | Q36968876 | ||
| A deletion in a photoreceptor-specific nuclear receptor mRNA causes retinal degeneration in the rd7 mouse | Q36981827 | ||
| Usher syndromes due to MYO7A, PCDH15, USH2A or GPR98 mutations share retinal disease mechanism | Q37323334 | ||
| Development of photoreceptor-specific promoters and their utility to investigate EIAV lentiviral vector mediated gene transfer to photoreceptors | Q38297007 | ||
| P275 | copyright license | Creative Commons Attribution 4.0 International | Q20007257 |
| P6216 | copyright status | copyrighted | Q50423863 |
| P4510 | describes a project that uses | ImageJ | Q1659584 |
| P433 | issue | 3 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | congenital disorder | Q727096 |
| Leber congenital amaurosis | Q1811132 | ||
| murine model | Q122890741 | ||
| P304 | page(s) | e92928 | |
| P577 | publication date | 2014-03-26 | |
| P1433 | published in | PLOS One | Q564954 |
| P1476 | title | Natural history of cone disease in the murine model of Leber congenital amaurosis due to CEP290 mutation: determining the timing and expectation of therapy | |
| P478 | volume | 9 |
| Q57456377 | A CEP290 C-Terminal Domain Complements the Mutant CEP290 of Rd16 Mice In Trans and Rescues Retinal Degeneration |
| Q40369908 | CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10. |
| Q91143094 | Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10 |
| Q36074089 | Gene Therapy Fully Restores Vision to the All-Cone Nrl(-/-) Gucy2e(-/-) Mouse Model of Leber Congenital Amaurosis-1. |
| Q36985099 | Identification and Correction of Mechanisms Underlying Inherited Blindness in Human iPSC-Derived Optic Cups. |
| Q39829369 | In vitro and in vivo rescue of aberrant splicing in CEP290-associated LCA by antisense oligonucleotide delivery |
| Q90152961 | Leber Congenital Amaurosis (LCA): Potential for Improvement of Vision |
| Q89975685 | Metabolic and Redox Signaling of the Nucleoredoxin-Like-1 Gene for the Treatment of Genetic Retinal Diseases |
| Q32186770 | Pupillary Light Reflexes in Severe Photoreceptor Blindness Isolate the Melanopic Component of Intrinsically Photosensitive Retinal Ganglion Cells. |
| Q36306263 | Retinal Gene Therapy: Current Progress and Future Prospects |
| Q37142915 | Retinal Structure and Gene Therapy Outcome in Retinoschisin-Deficient Mice Assessed by Spectral-Domain Optical Coherence Tomography |
| Q92850956 | Treatment Potential for Macular Cone Vision in Leber Congenital Amaurosis Due to CEP290 or NPHP5 Mutations: Predictions From Artificial Intelligence |
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