Let There Be Light: Gene and Cell Therapy for Blindness.

Stargardt disease: clinical features, molecular genetics, animal models and therapeutic options. Br J Ophthalmol (2016) 0.78

Molecular Diagnosis of Inherited Retinal Diseases in Indigenous African Populations by Whole-Exome Sequencing. Invest Ophthalmol Vis Sci (2016) 0.75

Applications of CRISPR/Cas9 in retinal degenerative diseases. Int J Ophthalmol (2017) 0.75

Three-dimensional retinal organoids from mouse pluripotent stem cells mimic in vivo development with enhanced stratification and rod photoreceptor differentiation. Mol Vis (2016) 0.75

Novel therapeutics for Stargardt disease. Graefes Arch Clin Exp Ophthalmol (2017) 0.75

Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell (2006) 120.51

Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell (2007) 101.42

Establishment in culture of pluripotential cells from mouse embryos. Nature (1981) 30.49

Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med (2008) 17.21

Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med (2008) 15.30

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. Hum Gene Ther (2008) 8.83

Embryonic stem cell trials for macular degeneration: a preliminary report. Lancet (2012) 7.70

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci U S A (2008) 7.16

Retinal repair by transplantation of photoreceptor precursors. Nature (2006) 6.73

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet (2009) 6.71

Leber congenital amaurosis: genes, proteins and disease mechanisms. Prog Retin Eye Res (2008) 5.90

Ectopic expression of a microbial-type rhodopsin restores visual responses in mice with photoreceptor degeneration. Neuron (2006) 5.65

Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial. Lancet (2014) 4.24

Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol Ther (2009) 3.93

Genetic reactivation of cone photoreceptors restores visual responses in retinitis pigmentosa. Science (2010) 3.92

Restoration of vision after transplantation of photoreceptors. Nature (2012) 3.91

Self-formation of optic cups and storable stratified neural retina from human ESCs. Cell Stem Cell (2012) 3.85

Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years. Arch Ophthalmol (2011) 3.79

Ocular immune privilege: therapeutic opportunities from an experiment of nature. Nat Rev Immunol (2003) 3.74

Light-activated channels targeted to ON bipolar cells restore visual function in retinal degeneration. Nat Neurosci (2008) 3.73

Ranibizumab therapy for neovascular age-related macular degeneration. N Engl J Med (2010) 3.71

Transplantation of human embryonic stem cell-derived photoreceptors restores some visual function in Crx-deficient mice. Cell Stem Cell (2009) 3.66

Ciliary neurotrophic factor (CNTF) for human retinal degeneration: phase I trial of CNTF delivered by encapsulated cell intraocular implants. Proc Natl Acad Sci U S A (2006) 3.49

Interim results from the international trial of Second Sight's visual prosthesis. Ophthalmology (2012) 3.35

Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or leber congenital amaurosis. Proc Natl Acad Sci U S A (1998) 3.22

Improvement and decline in vision with gene therapy in childhood blindness. N Engl J Med (2015) 3.16

High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Mol Ther (2008) 2.93

Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model. Hum Mol Genet (2001) 2.88

AAV2 gene therapy readministration in three adults with congenital blindness. Sci Transl Med (2012) 2.87

Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement. Proc Natl Acad Sci U S A (2013) 2.71

Long-term effect of gene therapy on Leber's congenital amaurosis. N Engl J Med (2015) 2.68

Identification and characterization of rod-derived cone viability factor. Nat Genet (2004) 2.63

A look at autoimmunity and inflammation in the eye. J Clin Invest (2010) 2.30

Compacted DNA nanoparticles administered to the nasal mucosa of cystic fibrosis subjects are safe and demonstrate partial to complete cystic fibrosis transmembrane regulator reconstitution. Hum Gene Ther (2004) 2.21

Rod-derived cone viability factor promotes cone survival by stimulating aerobic glycolysis. Cell (2015) 2.08

Photoreceptor precursors derived from three-dimensional embryonic stem cell cultures integrate and mature within adult degenerate retina. Nat Biotechnol (2013) 2.06

Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy. Gene Ther (2008) 2.00

Restoration of visual function in retinal degeneration mice by ectopic expression of melanopsin. Proc Natl Acad Sci U S A (2008) 1.89

In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous. Sci Transl Med (2013) 1.86

Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2. Ophthalmology (2013) 1.85

Repair of the degenerate retina by photoreceptor transplantation. Proc Natl Acad Sci U S A (2012) 1.79

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. Hum Mol Genet (2009) 1.76

Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous. Mol Ther (2009) 1.73

Generation of three-dimensional retinal tissue with functional photoreceptors from human iPSCs. Nat Commun (2014) 1.68

Artificial vision with wirelessly powered subretinal electronic implant alpha-IMS. Proc Biol Sci (2013) 1.63

Functional cone rescue by RdCVF protein in a dominant model of retinitis pigmentosa. Mol Ther (2009) 1.53

The human visual cortex responds to gene therapy-mediated recovery of retinal function. J Clin Invest (2011) 1.52

Restoration of Vision with Ectopic Expression of Human Rod Opsin. Curr Biol (2015) 1.51

Effective transplantation of photoreceptor precursor cells selected via cell surface antigen expression. Stem Cells (2011) 1.50

Stem Cell Therapies in Clinical Trials: Progress and Challenges. Cell Stem Cell (2015) 1.47

Optimized allotopic expression of the human mitochondrial ND4 prevents blindness in a rat model of mitochondrial dysfunction. Am J Hum Genet (2008) 1.43

Long-Term Results from an Epiretinal Prosthesis to Restore Sight to the Blind. Ophthalmology (2015) 1.42

Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse. PLoS One (2010) 1.33

Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors. PLoS One (2013) 1.33

Subretinal implantation of retinal pigment epithelial cells derived from human embryonic stem cells: improved survival when implanted as a monolayer. Invest Ophthalmol Vis Sci (2013) 1.30

Targeted transgene expression in muller glia of normal and diseased retinas using lentiviral vectors. Invest Ophthalmol Vis Sci (2007) 1.28

Transplantation of embryonic and induced pluripotent stem cell-derived 3D retinal sheets into retinal degenerative mice. Stem Cell Reports (2014) 1.22

Novel adeno-associated viral vectors for retinal gene therapy. Gene Ther (2011) 1.21

High-capacity adenoviral vectors for gene transfer and somatic gene therapy. Hum Gene Ther (1999) 1.20

Immune response following intraocular delivery of recombinant viral vectors. Gene Ther (2003) 1.20

Rod-derived cone viability factor for treating blinding diseases: from clinic to redox signaling. Sci Transl Med (2010) 1.20

Human retinal disease from AIPL1 gene mutations: foveal cone loss with minimal macular photoreceptors and rod function remaining. Invest Ophthalmol Vis Sci (2011) 1.19

DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice. J Clin Invest (2012) 1.19

Increased integration of transplanted CD73-positive photoreceptor precursors into adult mouse retina. Invest Ophthalmol Vis Sci (2011) 1.19

Mutation survey of known LCA genes and loci in the Saudi Arabian population. Invest Ophthalmol Vis Sci (2008) 1.15

CEP290 gene transfer rescues Leber congenital amaurosis cellular phenotype. Gene Ther (2014) 1.15

Vitreous: a barrier to nonviral ocular gene therapy. Invest Ophthalmol Vis Sci (2005) 1.14

Human RPE stem cells grown into polarized RPE monolayers on a polyester matrix are maintained after grafting into rabbit subretinal space. Stem Cell Reports (2014) 1.11

Transient photoreceptor deconstruction by CNTF enhances rAAV-mediated cone functional rescue in late stage CNGB3-achromatopsia. Mol Ther (2013) 1.10

AAV mediated GDNF secretion from retinal glia slows down retinal degeneration in a rat model of retinitis pigmentosa. Mol Ther (2011) 1.10

Gene therapy for blindness. Annu Rev Neurosci (2013) 1.09

Targeting channelrhodopsin-2 to ON-bipolar cells with vitreally administered AAV Restores ON and OFF visual responses in blind mice. Mol Ther (2014) 1.08

Restoring the ON Switch in Blind Retinas: Opto-mGluR6, a Next-Generation, Cell-Tailored Optogenetic Tool. PLoS Biol (2015) 1.04

From confluent human iPS cells to self-forming neural retina and retinal pigmented epithelium. Proc Natl Acad Sci U S A (2014) 1.04

Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno-associated virus capsid and promoter. EMBO Mol Med (2014) 1.04

AAV-mediated gene therapy in the guanylate cyclase (RetGC1/RetGC2) double knockout mouse model of Leber congenital amaurosis. Hum Gene Ther (2013) 1.03

Developing cellular therapies for retinal degenerative diseases. Invest Ophthalmol Vis Sci (2014) 1.03

Viral-mediated RdCVF and RdCVFL expression protects cone and rod photoreceptors in retinal degeneration. J Clin Invest (2014) 1.01

Cell replacement and visual restoration by retinal sheet transplants. Prog Retin Eye Res (2012) 1.01

Using human induced pluripotent stem cells to treat retinal disease. Prog Retin Eye Res (2013) 1.00

Cellular strategies for retinal repair by photoreceptor replacement. Prog Retin Eye Res (2015) 1.00

State-of-the-art lentiviral vectors for research use: risk assessment and biosafety recommendations. Curr Gene Ther (2009) 1.00

Stable and efficient intraocular gene transfer using pseudotyped EIAV lentiviral vectors. J Gene Med (2006) 0.99

Evaluation of Italian patients with leber congenital amaurosis due to AIPL1 mutations highlights the potential applicability of gene therapy. Invest Ophthalmol Vis Sci (2011) 0.99

Transplantation of human central nervous system stem cells - neuroprotection in retinal degeneration. Eur J Neurosci (2012) 0.99

Intravitreal delivery of AAV-NDI1 provides functional benefit in a murine model of Leber hereditary optic neuropathy. Eur J Hum Genet (2012) 0.98

Long-term transgene expression in the RPE after gene transfer with a high-capacity adenoviral vector. Invest Ophthalmol Vis Sci (2002) 0.98

Ocular gene therapy: a review of nonviral strategies. Mol Vis (2006) 0.98

Derivation of traceable and transplantable photoreceptors from mouse embryonic stem cells. Stem Cell Reports (2014) 0.97

Gene Therapy for Leber Hereditary Optic Neuropathy: Initial Results. Ophthalmology (2015) 0.97

Comparative analysis of DNA nanoparticles and AAVs for ocular gene delivery. PLoS One (2012) 0.96

Transduction of photoreceptors with equine infectious anemia virus lentiviral vectors: safety and biodistribution of StarGen for Stargardt disease. Invest Ophthalmol Vis Sci (2013) 0.95

Synaptic pathology and therapeutic repair in adult retinoschisis mouse by AAV-RS1 transfer. J Clin Invest (2015) 0.95

Retinal cell type expression specificity of HIV-1-derived gene transfer vectors upon subretinal injection in the adult rat: influence of pseudotyping and promoter. J Gene Med (2005) 0.94

The optimized allotopic expression of ND1 or ND4 genes restores respiratory chain complex I activity in fibroblasts harboring mutations in these genes. Biochim Biophys Acta (2008) 0.94

Five recombinant simian immunodeficiency virus pseudotypes lead to exclusive transduction of retinal pigmented epithelium in rat. Mol Ther (2002) 0.93

Halting progressive neurodegeneration in advanced retinitis pigmentosa. J Clin Invest (2015) 0.93

Adenovirus vectors targeting distinct cell types in the retina. Invest Ophthalmol Vis Sci (2009) 0.92

Equine infectious anemia viral vector-mediated codelivery of endostatin and angiostatin driven by retinal pigmented epithelium-specific VMD2 promoter inhibits choroidal neovascularization. Hum Gene Ther (2009) 0.92

Immune responses limit adenovirally mediated gene expression in the adult mouse eye. Gene Ther (1998) 0.92