| scholarly article | Q13442814 |
| P50 | author | Astra Dinculescu | Q48237543 |
| P2093 | author name string | Felix Tonagel | |
| Naoyuki Tanimoto | |||
| Mathias W Seeliger | |||
| Bernhard H F Weber | |||
| William W Hauswirth | |||
| Astra Dinculescu | |||
| Adrian M Timmers | |||
| Robert S Molday | |||
| Laurie L Molday | |||
| Andreas Janssen | |||
| Seok H Min | |||
| P2860 | cites work | Positional cloning of the gene associated with X-linked juvenile retinoschisis | Q24317256 |
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| Photoreceptor peripherin is the normal product of the gene responsible for retinal degeneration in the rds mouse | Q24564633 | ||
| Defective Discoidin Domain Structure, Subunit Assembly, and Endoplasmic Reticulum Processing of Retinoschisin are Primary Mechanisms Responsible for X-linked Retinoschisis | Q28204024 | ||
| Intracellular retention of mutant retinoschisin is the pathological mechanism underlying X-linked retinoschisis | Q28213609 | ||
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| RS-1 Gene Delivery to an Adult Rs1h Knockout Mouse Model Restores ERG b-Wave with Reversal of the Electronegative Waveform of X-Linked Retinoschisis | Q28506399 | ||
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| Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity | Q39686097 | ||
| New views on RPE65 deficiency: the rod system is the source of vision in a mouse model of Leber congenital amaurosis. | Q43723701 | ||
| Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model | Q43829645 | ||
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| Glial fibrillary acidic protein (GFAP) is synthesized in the early stages of the photoreceptor cell degeneration of the mivit/mivit (vitiligo) mouse | Q73519377 | ||
| P433 | issue | 4 | |
| P921 | main subject | gene therapy | Q213901 |
| P304 | page(s) | 644-651 | |
| P577 | publication date | 2005-10-01 | |
| P1433 | published in | Molecular Therapy | Q15762400 |
| P1476 | title | Prolonged recovery of retinal structure/function after gene therapy in an Rs1h-deficient mouse model of x-linked juvenile retinoschisis | |
| P478 | volume | 12 |
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| Q29465780 | Clinical and genetic findings in Hungarian patients with X-linked juvenile retinoschisis |
| Q46940069 | Comparative analysis of in vivo and in vitro AAV vector transduction in the neonatal mouse retina: effects of serotype and site of administration. |
| Q38535037 | Convergence of Human Genetics and Animal Studies: Gene Therapy for X-Linked Retinoschisis |
| Q27022731 | Dawn of ocular gene therapy: implications for molecular diagnosis in retinal disease |
| Q44690695 | Effect of late-stage therapy on disease progression in AAV-mediated rescue of photoreceptor cells in the retinoschisin-deficient mouse |
| Q36236887 | Elevation of intraocular pressure in rodents using viral vectors targeting the trabecular meshwork |
| Q39013159 | Gene and cell-based therapies for inherited retinal disorders: An update |
| Q40820678 | Gene delivery to the retina: from mouse to man. |
| Q37950225 | Gene supplementation therapy for recessive forms of inherited retinal dystrophies. |
| Q38221905 | Gene therapies for inherited retinal disorders. |
| Q41465719 | Gene therapy as a treatment concept for inherited retinal diseases |
| Q36534765 | Gene therapy progress and prospects: the eye. |
| Q36515706 | Genetic modification of the schisis phenotype in a mouse model of X-linked retinoschisis |
| Q33713361 | High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors |
| Q27023474 | Immunology of AAV-Mediated Gene Transfer in the Eye |
| Q33224068 | In vivo confocal imaging of the retina in animal models using scanning laser ophthalmoscopy |
| Q37096060 | Inherited diseases of photoreceptors and prospects for gene therapy. |
| Q38214595 | Insights gained from gene therapy in animal models of retGC1 deficiency |
| Q37414094 | Intravitreal delivery of AAV8 retinoschisin results in cell type-specific gene expression and retinal rescue in the Rs1-KO mouse |
| Q38254324 | Leber congenital amaurosis caused by mutations in GUCY2D. |
| Q34382138 | Long-term 12 year follow-up of X-linked congenital retinoschisis |
| Q30352027 | Molecular modeling indicates distinct classes of missense variants with mild and severe XLRS phenotypes |
| Q30384232 | Molecular modeling of retinoschisin with functional analysis of pathogenic mutations from human X-linked retinoschisis. |
| Q30160010 | Molecular pathology of X linked retinoschisis: mutations interfere with retinoschisin secretion and oligomerisation |
| Q37057166 | Mouse Models of NMNAT1-Leber Congenital Amaurosis (LCA9) Recapitulate Key Features of the Human Disease |
| Q37284890 | Nonviral ocular gene therapy: assessment and future directions |
| Q28708927 | Novel RS1 mutations associated with X-linked juvenile retinoschisis |
| Q37944999 | Novel adeno-associated viral vectors for retinal gene therapy |
| Q36099346 | Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors |
| Q37225183 | Ocular and systemic safety of a recombinant AAV8 vector for X-linked retinoschisis gene therapy: GLP studies in rabbits and Rs1-KO mice |
| Q36825150 | Preclinical Dose-Escalation Study of Intravitreal AAV-RS1 Gene Therapy in a Mouse Model of X-linked Retinoschisis: Dose-Dependent Expression and Improved Retinal Structure and Function |
| Q60309662 | Prospective Evaluation of Patients With X-Linked Retinoschisis During 18 Months |
| Q45882673 | Prospects for gene therapy of inherited retinal disease |
| Q37153811 | RD3 gene delivery restores guanylate cyclase localization and rescues photoreceptors in the Rd3 mouse model of Leber congenital amaurosis 12. |
| Q34631983 | Recombinant vectors based on porcine adeno-associated viral serotypes transduce the murine and pig retina |
| Q34451693 | Reduction of choroidal neovascularization in mice by adeno-associated virus-delivered anti-vascular endothelial growth factor short hairpin RNA. |
| Q37968572 | Regenerative nanomedicine and the treatment of degenerative retinal diseases |
| Q33954586 | Regulation of retinoschisin secretion in Weri-Rb1 cells by the F-actin and microtubule cytoskeleton |
| Q36951825 | Rescue of sight by gene therapy - closer than it may appear |
| Q41651450 | Restoration of synaptic function in sight for degenerative retinal disease |
| Q37142915 | Retinal Structure and Gene Therapy Outcome in Retinoschisin-Deficient Mice Assessed by Spectral-Domain Optical Coherence Tomography |
| Q37955772 | Retinal dystrophies and gene therapy. |
| Q42007962 | Retinal gene therapy using adeno-associated viral vectors: multiple applications for a small virus. |
| Q21203028 | Retinitis pigmentosa |
| Q29465531 | Retinoschisin (RS1) interacts with negatively charged lipid bilayers in the presence of Ca2+: an atomic force microscopy study |
| Q40362704 | Retinoschisin gene therapy in photoreceptors, Müller glia or all retinal cells in the Rs1h-/- mouse. |
| Q36677162 | Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin |
| Q30847836 | Structural analysis of X-linked retinoschisis mutations reveals distinct classes which differentially effect retinoschisin function. |
| Q27319838 | Structure-function correlation of the human central retina |
| Q34497784 | Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease |
| Q36040298 | Synaptic pathology and therapeutic repair in adult retinoschisis mouse by AAV-RS1 transfer |
| Q36915061 | Synaptic pathology in retinoschisis knockout (Rs1-/y) mouse retina and modification by rAAV-Rs1 gene delivery |
| Q34296856 | Test-Retest Intervisit Variability of Functional and Structural Parameters in X-Linked Retinoschisis |
| Q38337392 | The Status of RPE65 Gene Therapy Trials: Safety and Efficacy |
| Q33433853 | The effects of transient retinal detachment on cavity size and glial and neural remodeling in a mouse model of X-linked retinoschisis |
| Q30525669 | The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina. |
| Q37351985 | The role of the photoreceptor ABC transporter ABCA4 in lipid transport and Stargardt macular degeneration |
| Q36448393 | Treatment of human disease by adeno-associated viral gene transfer |
| Q36844127 | Truncation of retinoschisin protein associated with a novel splice site mutation in the RS1 gene. |
| Q36965152 | Versatility of AAV vectors for retinal gene transfer |
| Q36492248 | Wide-field spectral-domain optical coherence tomography in patients and carriers of X-linked retinoschisis |
| Q29465813 | Wild-type and missense mutants of retinoschisin co-assemble resulting in either intracellular retention or incorrect assembly of the functionally active octamer. |
| Q35907087 | X-linked juvenile retinoschisis: clinical diagnosis, genetic analysis, and molecular mechanisms. |
| Q24647978 | X-linked retinoschisis: an update |
| Q34768118 | shRNA knockdown of guanylate cyclase 2e or cyclic nucleotide gated channel alpha 1 increases photoreceptor survival in a cGMP phosphodiesterase mouse model of retinitis pigmentosa |
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