| scholarly article | Q13442814 |
| P50 | author | James W. Bainbridge | Q42292899 |
| P2093 | author name string | Xiaoqing Liu | |
| Xiaoyun Xu | |||
| Basil Pawlyk | |||
| Tiansen Li | |||
| Robin R Ali | |||
| Alexander J Smith | |||
| Hoai Viet Tran | |||
| Jenny McIntosh | |||
| Amit Nathwani | |||
| Mark Basche | |||
| Mei Hong Tan | |||
| P2860 | cites work | Prevalence of AIPL1 mutations in inherited retinal degenerative disease | Q55670727 |
| Effects of low AIPL1 expression on phototransduction in rods | Q83202005 | ||
| AIPL1, a protein implicated in Leber's congenital amaurosis, interacts with and aids in processing of farnesylated proteins | Q24297621 | ||
| The inherited blindness associated protein AIPL1 interacts with the cell cycle regulator protein NUB1 | Q24310496 | ||
| AIPL1, the protein that is defective in Leber congenital amaurosis, is essential for the biosynthesis of retinal rod cGMP phosphodiesterase | Q24564587 | ||
| Safety and efficacy of gene transfer for Leber's congenital amaurosis | Q24634724 | ||
| Recombinant adeno-associated virus type 2-mediated gene delivery into the Rpe65-/- knockout mouse eye results in limited rescue | Q24802168 | ||
| Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis. | Q24811718 | ||
| Effect of gene therapy on visual function in Leber's congenital amaurosis | Q28277981 | ||
| Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy | Q28279907 | ||
| Retinal-specific guanylate cyclase gene mutations in Leber's congenital amaurosis | Q28298481 | ||
| Leber congenital amaurosis linked to AIPL1: a mouse model reveals destabilization of cGMP phosphodiesterase | Q28509144 | ||
| The Leber congenital amaurosis gene product AIPL1 is localized exclusively in rod photoreceptors of the adult human retina | Q30051782 | ||
| Leber congenital amaurosis | Q33754971 | ||
| Building a better vector: the manipulation of AAV virions | Q33928283 | ||
| The phenotype of Leber congenital amaurosis in patients with AIPL1 mutations | Q34332943 | ||
| The Leber congenital amaurosis protein AIPL1 modulates the nuclear translocation of NUB1 and suppresses inclusion formation by NUB1 fragments | Q34345764 | ||
| Unfolding retinal dystrophies: a role for molecular chaperones? | Q34351471 | ||
| The role of chaperone proteins in the aryl hydrocarbon receptor core complex | Q34815714 | ||
| Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates | Q35628868 | ||
| A null mutation in the photoreceptor guanylate cyclase gene causes the retinal degeneration chicken phenotype | Q35803466 | ||
| Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors | Q36099346 | ||
| AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDEbeta mutation | Q36678695 | ||
| Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness | Q36825024 | ||
| Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics | Q36937189 | ||
| Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer | Q39551137 | ||
| From mice to men: the cyclic GMP phosphodiesterase gene in vision and disease. The Proctor Lecture. | Q40584439 | ||
| High-titer recombinant adeno-associated virus production from replicating amplicons and herpes vectors deleted for glycoprotein H. | Q40791512 | ||
| Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy. | Q40869164 | ||
| Encapsidated adenovirus mini-chromosome-mediated delivery of genes to the retina: application to the rescue of photoreceptor degeneration | Q40995318 | ||
| Gene therapy restores vision in a canine model of childhood blindness | Q43590733 | ||
| AAV-Mediated gene transfer slows photoreceptor loss in the RCS rat model of retinitis pigmentosa | Q44541941 | ||
| Long-term evaluation of retinal function in Prph2Rd2/Rd2 mice following AAV-mediated gene replacement therapy | Q44569755 | ||
| Rescue of photoreceptor function by AAV-mediated gene transfer in a mouse model of inherited retinal degeneration | Q44933268 | ||
| Long-term preservation of retinal function in the RCS rat model of retinitis pigmentosa following lentivirus-mediated gene therapy | Q45230950 | ||
| Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8. | Q45399272 | ||
| Gene transfer into the mouse retina mediated by an adeno-associated viral vector | Q45877692 | ||
| Gene replacement therapy rescues photoreceptor degeneration in a murine model of Leber congenital amaurosis lacking RPGRIP. | Q45886246 | ||
| Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis | Q45887612 | ||
| Mental retardation in amaurosis congenita of Leber. | Q51987220 | ||
| P433 | issue | 12 | |
| P921 | main subject | gene therapy | Q213901 |
| congenital disorder | Q727096 | ||
| retinitis pigmentosa | Q847057 | ||
| Leber congenital amaurosis | Q1811132 | ||
| P304 | page(s) | 2099-2114 | |
| P577 | publication date | 2009-03-19 | |
| P1433 | published in | Human Molecular Genetics | Q2720965 |
| P1476 | title | Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors | |
| P478 | volume | 18 |
| Q35766381 | A long-term efficacy study of gene replacement therapy for RPGR-associated retinal degeneration |
| Q27009291 | AAV-mediated gene therapy in mouse models of recessive retinal degeneration |
| Q39007935 | AAV8(Y733F)-mediated gene therapy in a Spata7 knockout mouse model of Leber congenital amaurosis and retinitis pigmentosa |
| Q45875371 | Absence of ocular malignant transformation after sub-retinal delivery of rAAV2/2 or integrating lentiviral vectors in p53-deficient mice |
| Q37190609 | Advances in Gene Therapy for Diseases of the Eye. |
| Q36146011 | Application of a high-throughput genotyping method for loci exclusion in non-consanguineous Australian pedigrees with autosomal recessive retinitis pigmentosa |
| Q28084726 | Biology and therapy of inherited retinal degenerative disease: insights from mouse models |
| Q35603676 | C1q enhances cone photoreceptor survival in a mouse model of autosomal recessive retinitis pigmentosa |
| Q37963356 | Clinical and Rehabilitative Management of Retinitis Pigmentosa: Up-to-Date |
| Q35863012 | Correlation of structure and function of the macula in patients with retinitis pigmentosa |
| Q42105112 | Current concepts in the treatment of retinitis pigmentosa |
| Q41087457 | Differential effects of unfolded protein response pathways on axon injury-induced death of retinal ganglion cells. |
| Q35804801 | Differential targeting of feline photoreceptors by recombinant adeno-associated viral vectors: implications for preclinical gene therapy trials. |
| Q33862495 | Dominant cone-rod dystrophy: a mouse model generated by gene targeting of the GCAP1/Guca1a gene |
| Q33668644 | Early alteration of retinal neurons in Aipl1-/- animals. |
| Q42235082 | Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors |
| Q33622429 | Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse. |
| Q40820678 | Gene delivery to the retina: from mouse to man. |
| Q37950225 | Gene supplementation therapy for recessive forms of inherited retinal dystrophies. |
| Q34994553 | Gene therapeutic approach using mutation-adapted U1 snRNA to correct a RPGR splice defect in patient-derived cells |
| Q38221905 | Gene therapies for inherited retinal disorders. |
| Q38014265 | Gene therapy for Leber congenital amaurosis: advances and future directions. |
| Q38073020 | Gene therapy for retinal disease |
| Q36147481 | Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients |
| Q30626026 | Gene therapy restores vision in rd1 mice after removal of a confounding mutation in Gpr179 |
| Q35532922 | Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis |
| Q33584461 | Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations |
| Q36598421 | Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement |
| Q38214595 | Insights gained from gene therapy in animal models of retGC1 deficiency |
| Q34937075 | Intravitreal injection of AAV2 transduces macaque inner retina |
| Q34193092 | Leber congenital amaurosis associated with AIPL1: challenges in ascribing disease causation, clinical findings, and implications for gene therapy. |
| Q38254324 | Leber congenital amaurosis caused by mutations in GUCY2D. |
| Q39423311 | Leber congenital amaurosis/early-onset severe retinal dystrophy: clinical features, molecular genetics and therapeutic interventions |
| Q36654534 | Let There Be Light: Gene and Cell Therapy for Blindness |
| Q37784661 | Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies |
| Q35119686 | Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy |
| Q35518971 | Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse. |
| Q38899558 | Long-term preservation of cones and improvement in visual function following gene therapy in a mouse model of leber congenital amaurosis caused by guanylate cyclase-1 deficiency. |
| Q97516701 | Loss of Arid1a Promotes Neuronal Survival Following Optic Nerve Injury |
| Q37452605 | Molecular characterization of retinitis pigmentosa in Saudi Arabia |
| Q39696935 | Multiple components in serum contribute to hepatic transgene expression by lipoplex in mice |
| Q34061577 | Mutation screening of retinal dystrophy patients by targeted capture from tagged pooled DNAs and next generation sequencing |
| Q42032781 | Mutation-independent rescue of a novel mouse model of Retinitis Pigmentosa |
| Q42729265 | Non-erythropoietic erythropoietin derivatives protect from light-induced and genetic photoreceptor degeneration. |
| Q35169723 | Novel C8orf37 mutations cause retinitis pigmentosa in consanguineous families of Pakistani origin |
| Q37944999 | Novel adeno-associated viral vectors for retinal gene therapy |
| Q39521004 | Pharmacological enhancement of ex vivo gene therapy neuroprotection in a rodent model of retinal degeneration |
| Q36890858 | Photoreceptor rescue by an abbreviated human RPGR gene in a murine model of X-linked retinitis pigmentosa. |
| Q27007079 | Promising and delivering gene therapies for vision loss |
| Q39546264 | Quantifying transduction efficiencies of unmodified and tyrosine capsid mutant AAV vectors in vitro using two ocular cell lines |
| Q34614424 | RNAi-mediated gene suppression in a GCAP1(L151F) cone-rod dystrophy mouse model |
| Q34631983 | Recombinant vectors based on porcine adeno-associated viral serotypes transduce the murine and pig retina |
| Q34085940 | Replacement gene therapy with a human RPGRIP1 sequence slows photoreceptor degeneration in a murine model of Leber congenital amaurosis |
| Q35522527 | Residual electroretinograms in young Leber congenital amaurosis patients with mutations of AIPL1 |
| Q37466380 | Restoration of vision after transplantation of photoreceptors |
| Q90664456 | Retinal Organoids derived from hiPSCs of an AIPL1-LCA Patient Maintain Cytoarchitecture despite Reduced levels of Mutant AIPL1 |
| Q35050757 | Review and update on the molecular basis of Leber congenital amaurosis |
| Q34497784 | Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease |
| Q30571313 | Successful gene therapy in the RPGRIP1-deficient dog: a large model of cone-rod dystrophy |
| Q33697709 | The Leber congenital amaurosis protein, AIPL1, is needed for the viability and functioning of cone photoreceptor cells |
| Q30525669 | The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina. |
| Q37716561 | Tyrosine-mutant AAV8 delivery of human MERTK provides long-term retinal preservation in RCS rats. |
| Q34561531 | Vector platforms for gene therapy of inherited retinopathies. |
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