Published in Gene Ther on October 05, 2006
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
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
The AAV vector toolkit: poised at the clinical crossroads. Mol Ther (2012) 2.53
Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors. J Virol (2007) 2.05
Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy. Gene Ther (2008) 2.00
The retinal pigment epithelium in health and disease. Curr Mol Med (2010) 1.87
Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy. Prog Retin Eye Res (2010) 1.81
Canine and human visual cortex intact and responsive despite early retinal blindness from RPE65 mutation. PLoS Med (2007) 1.79
A comprehensive review of retinal gene therapy. Mol Ther (2013) 1.70
RPE65: role in the visual cycle, human retinal disease, and gene therapy. Ophthalmic Genet (2009) 1.39
Topographical characterization of cone photoreceptors and the area centralis of the canine retina. Mol Vis (2008) 1.23
Detection of intact rAAV particles up to 6 years after successful gene transfer in the retina of dogs and primates. Mol Ther (2008) 1.20
Advances in gene therapy technologies to treat retinitis pigmentosa. Clin Ophthalmol (2013) 1.10
Gene therapy in the second eye of RPE65-deficient dogs improves retinal function. Gene Ther (2010) 1.09
Gene therapy for retinal disease. Transl Res (2013) 1.03
Evaluation of a behavioral method for objective vision testing and identification of achromatopsia in dogs. Am J Vet Res (2010) 0.97
Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach. Gene Ther (2013) 0.97
Histopathology and functional correlations in a patient with a mutation in RPE65, the gene for retinol isomerase. Invest Ophthalmol Vis Sci (2011) 0.95
Gene therapy for Leber congenital amaurosis: advances and future directions. Graefes Arch Clin Exp Ophthalmol (2012) 0.95
Vector platforms for gene therapy of inherited retinopathies. Prog Retin Eye Res (2014) 0.94
Evading the immune response upon in vivo gene therapy with viral vectors. Curr Opin Mol Ther (2009) 0.94
The use of canine models of inherited retinal degeneration to test novel therapeutic approaches. Vet Ophthalmol (2009) 0.94
An analysis of retinal gene therapy clinical trials. Curr Opin Mol Ther (2009) 0.93
The RPGRIP1-deficient dog, a promising canine model for gene therapy. Mol Vis (2009) 0.92
Gene therapy for ocular diseases. Br J Ophthalmol (2010) 0.90
Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients. Hum Gene Ther (2012) 0.90
Barriers for retinal gene therapy: separating fact from fiction. Vision Res (2008) 0.88
Gene therapy of inherited retinal degenerations: prospects and challenges. Hum Gene Ther (2015) 0.87
Gene therapy regenerates protein expression in cone photoreceptors in Rpe65(R91W/R91W) mice. PLoS One (2011) 0.86
A review of therapeutic prospects of non-viral gene therapy in the retinal pigment epithelium. Biomaterials (2013) 0.85
Promising and delivering gene therapies for vision loss. Vision Res (2014) 0.85
Differential proteomics and functional research following gene therapy in a mouse model of Leber congenital amaurosis. PLoS One (2012) 0.82
The genetics of eye disorders in the dog. Canine Genet Epidemiol (2014) 0.82
Progressive retinal atrophy in the Border Collie: a new XLPRA. BMC Vet Res (2008) 0.81
Suprachoroidal electrotransfer: a nonviral gene delivery method to transfect the choroid and the retina without detaching the retina. Mol Ther (2012) 0.81
Gene Therapy of ABCA4-Associated Diseases. Cold Spring Harb Perspect Med (2015) 0.81
Regulation of retinal function but nonrescue of vision in RPE65-deficient dogs treated with doxycycline-regulatable AAV vectors. Mol Ther (2010) 0.80
AAV and compacted DNA nanoparticles for the treatment of retinal disorders: challenges and future prospects. Invest Ophthalmol Vis Sci (2011) 0.80
Dog models for blinding inherited retinal dystrophies. Hum Gene Ther Clin Dev (2015) 0.80
A single intravenous AAV9 injection mediates bilateral gene transfer to the adult mouse retina. PLoS One (2013) 0.80
A 350 bp region of the proximal promoter of Rds drives cell-type specific gene expression. Exp Eye Res (2010) 0.79
In vivo evaluation of the cornea and conjunctiva of the normal laboratory beagle using time- and Fourier-domain optical coherence tomography and ultrasound pachymetry. Vet Ophthalmol (2015) 0.79
Impacts of nanomedicines in ocular pharmacotherapy. Bioimpacts (2011) 0.79
Sustained gene expression in the retina by improved episomal vectors. Tissue Eng Part A (2014) 0.79
Immunoregulatory function of IL-27 and TGF-β1 in cardiac allograft transplantation. Transplantation (2012) 0.78
Retinal Gene Therapy: Current Progress and Future Prospects. Expert Rev Ophthalmol (2015) 0.78
Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids. Methods Mol Biol (2016) 0.77
Republished review: Gene therapy for ocular diseases. Postgrad Med J (2011) 0.77
Homozygosity mapping and targeted sanger sequencing reveal genetic defects underlying inherited retinal disease in families from pakistan. PLoS One (2015) 0.77
Immuno-histochemical analysis of rod and cone reaction to RPE65 deficiency in the inferior and superior canine retina. PLoS One (2014) 0.76
Advances in Gene Therapy for Diseases of the Eye. Hum Gene Ther (2016) 0.75
Retinal Gene Therapy: Surgical Vector Delivery in the Translation to Clinical Trials. Front Neurosci (2017) 0.75
Clinical Trials in Retinal Dystrophies. Middle East Afr J Ophthalmol (2016) 0.75
Gene therapy approaches for the treatment of retinal disorders. Discov Med (2016) 0.75
Outer Plexiform Layer Structures Are Not Altered Following AAV-Mediated Gene Transfer in Healthy Rat Retina. Front Neurol (2017) 0.75
"To perpetuate blindness!": attitudes of UK patients with inherited retinal disease towards genetic testing. J Community Genet (2013) 0.75
Cloning in single-stranded bacteriophage as an aid to rapid DNA sequencing. J Mol Biol (1980) 61.16
Sequence and organization of the human mitochondrial genome. Nature (1981) 57.39
Size and charge isomer separation and estimation of molecular weights of proteins by disc gel electrophoresis. Arch Biochem Biophys (1968) 15.05
Quality of hospital care for seriously ill children in less-developed countries. Lancet (2001) 7.08
Retinal repair by transplantation of photoreceptor precursors. Nature (2006) 6.73
Transferable vancomycin and teicoplanin resistance in Enterococcus faecium. Antimicrob Agents Chemother (1989) 5.31
Epidemiology of ischemic stroke subtypes according to TOAST criteria: incidence, recurrence, and long-term survival in ischemic stroke subtypes: a population-based study. Stroke (2001) 4.87
DNA sequence analysis by primed synthesis. Methods Enzymol (1980) 4.75
Biochemical basis of obligate autotrophy in blue-green algae and thiobacilli. J Bacteriol (1967) 4.63
The oncogenic cysteine-rich LIM domain protein rbtn2 is essential for erythroid development. Cell (1994) 4.58
Time for a review of admission to medical school? Lancet (1995) 4.56
A dust-obscured massive maximum-starburst galaxy at a redshift of 6.34. Nature (2013) 4.43
Randomised trial of Haemophilus influenzae type-b tetanus protein conjugate vaccine [corrected] for prevention of pneumonia and meningitis in Gambian infants. Lancet (1997) 4.32
Nitrogen chlorosis in blue-green algae. Arch Mikrobiol (1969) 4.18
Restoration of vision after transplantation of photoreceptors. Nature (2012) 3.91
A method for sequencing restriction fragments with dideoxynucleoside triphosphates. Nucleic Acids Res (1978) 3.90
B cell ligand discrimination through a spreading and contraction response. Science (2006) 3.89
Different pattern of codon recognition by mammalian mitochondrial tRNAs. Proc Natl Acad Sci U S A (1980) 3.74
Beta-adrenergic activation and memory for emotional events. Nature (1994) 3.62
Assessment of inactivated influenza-A vaccine after three outbreaks of influenza A at Christ's Hospital. Lancet (1979) 3.55
A potent synthetic peptide inhibitor of the cAMP-dependent protein kinase. J Biol Chem (1986) 3.49
Feasibility and efficacy of routine PCR screening of blood donations for hepatitis C virus, hepatitis B virus, and HIV-1 in a blood-bank setting. Lancet (1999) 3.17
Dislocation rates of perineural catheters: a volunteer study. Br J Anaesth (2013) 3.05
Characterization of novel safe lentiviral vectors derived from simian immunodeficiency virus (SIVmac251) that efficiently transduce mature human dendritic cells. Gene Ther (2000) 2.95
Energy release in the solar corona from spatially resolved magnetic braids. Nature (2013) 2.90
Altered nociception, analgesia and aggression in mice lacking the receptor for substance P. Nature (1998) 2.90
Dexmedetomidine as an adjuvant to ropivacaine prolongs peripheral nerve block: a volunteer study. Br J Anaesth (2012) 2.89
MDR1 P-glycoprotein is a lipid translocase of broad specificity, while MDR3 P-glycoprotein specifically translocates phosphatidylcholine. Cell (1996) 2.87
Search for a resonance decaying into WZ boson pairs in pp collisions. Phys Rev Lett (2010) 2.83
Temperature-dependent alterations in sugar transport in cells infected by a temperature-sensitive mutant of Rous sarcoma virus. Proc Natl Acad Sci U S A (1971) 2.83
Acetate assimilation by Nitrobacter agilis in relation to its "obligate autotrophy". J Bacteriol (1968) 2.74
Warm-coding deficits and aberrant inflammatory pain in mice lacking P2X3 receptors. Nature (2000) 2.73
X-linked Alport syndrome: natural history in 195 families and genotype- phenotype correlations in males. J Am Soc Nephrol (2000) 2.71
[Determining the current position regarding the value of pioglitazone for the therapy of diabetes]. Dtsch Med Wochenschr (2007) 2.67
Two genetically separable steps in the differentiation of thymic epithelium. Science (1996) 2.66
Homologous recombination between defective neo genes in mouse 3T6 cells. Cold Spring Harb Symp Quant Biol (1984) 2.65
Evidence for Alfvén waves in solar x-ray jets. Science (2007) 2.63
Large-scale purification of the acetylcholine-receptor protein in its membrane-bound and detergent-extracted forms from Torpedo marmorata electric organ. Eur J Biochem (1977) 2.59
Antibiotic prescribing for sinusitis? Br Dent J (2000) 2.55
Saphenous vein puncture for blood sampling of the mouse, rat, hamster, gerbil, guinea pig, ferret and mink. Lab Anim (1998) 2.51
The use of exonuclease III for preparing single stranded DNA for use as a template in the chain terminator sequencing method. Nucleic Acids Res (1979) 2.51
Endoglin, an ancillary TGFbeta receptor, is required for extraembryonic angiogenesis and plays a key role in heart development. Dev Biol (2000) 2.50
Self-poisoning with drugs: a worsening situation. Br Med J (1972) 2.43
Determination of debrisoquine and its 4-hydroxy metabolite in biological fluids by gas chromatography with flame-ionization and nitrogen-selective detection. J Chromatogr (1977) 2.43
Identification of a penta- and hexapeptide of islet amyloid polypeptide (IAPP) with amyloidogenic and cytotoxic properties. J Mol Biol (2000) 2.34
A virus from epidemic vomiting disease. Br Med J (1972) 2.26
Submillimetre galaxies reside in dark matter haloes with masses greater than 3 × 10(11) solar masses. Nature (2011) 2.21
Randomized clinical trial comparing venous cutdown with the Seldinger technique for placement of implantable venous access ports. Br J Surg (2009) 2.18
A new redox cofactor in eukaryotic enzymes: 6-hydroxydopa at the active site of bovine serum amine oxidase. Science (1990) 2.16
Intraoperative neurophysiologic monitoring in spine surgery. Developments and state of the art in France in 2011. Orthop Traumatol Surg Res (2013) 2.15
Purification from Torpedo marmorata electric tissue of membrane fragments particularly rich in cholinergic receptor protein. FEBS Lett (1972) 2.09
Ligand-gated ion channel subunit partnerships: GABAA receptor alpha6 subunit gene inactivation inhibits delta subunit expression. J Neurosci (1997) 2.08
A crosslinked cofactor in lysyl oxidase: redox function for amino acid side chains. Science (1996) 2.06
Inhibition of tumor necrosis factor-alpha release in rat experimental endotoxemia by treatment with the 21-aminosteroid U-74389G. Crit Care Med (1999) 2.06
CpG ODN-induced matrix metalloproteinase-13 expression is mediated via activation of the ERK and NF-κB signalling pathways in odontoblast cells. Int Endod J (2013) 2.03
Limitations of sniff nasal pressure as an outcome measurement in amyotrophic lateral sclerosis patients in a clinical trial. Respiration (2012) 2.02
Stop paradigm erosion. J Manipulative Physiol Ther (1991) 2.00
Influenza A and meningococcal disease. Lancet (1991) 2.00
Impact of a short course in pharmacotherapy for undergraduate medical students: an international randomised controlled study. Lancet (1995) 1.97
Influenza at Christ's Hospital: March, 1974. Lancet (1976) 1.96
Glucose induces beta-cell apoptosis via upregulation of the Fas receptor in human islets. Diabetes (2001) 1.95
Laparoscopic nephrectomy: the experience of the laparoscopy working group of the German Urologic Association. J Urol (1998) 1.93
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. Gene Ther (2001) 1.93
Why does Australia have no national drug policy? Med J Aust (1992) 1.92
The mll-AF9 gene fusion in mice controls myeloproliferation and specifies acute myeloid leukaemogenesis. EMBO J (1999) 1.88
The important role of neuropeptides in complex regional pain syndrome. Neurology (2001) 1.88
Factors influencing recombinant adeno-associated virus production. Hum Gene Ther (1998) 1.86
Cloning and expression of cyclosporin A- and FK506-sensitive nuclear factor of activated T-cells: NF45 and NF90. J Biol Chem (1994) 1.85
Humor as a character strength among the elderly: empirical findings on age-related changes and its contribution to satisfaction with life. Z Gerontol Geriatr (2009) 1.84
CD45-null transgenic mice reveal a positive regulatory role for CD45 in early thymocyte development, in the selection of CD4+CD8+ thymocytes, and B cell maturation. J Exp Med (1996) 1.83
Analysis of the expression of MRP, the gene for a new putative transmembrane drug transporter, in human multidrug resistant lung cancer cell lines. Cancer Res (1993) 1.81
Protective immunity induced by Bacillus anthracis toxin-deficient strains. Infect Immun (1995) 1.81
Biogenesis and intranuclear trafficking of human box C/D and H/ACA RNPs. Cold Spring Harb Symp Quant Biol (2006) 1.80
Designing new treatment strategies in vital pulp therapy. J Dent (2000) 1.80
Pharmacological disruption of the outer limiting membrane leads to increased retinal integration of transplanted photoreceptor precursors. Exp Eye Res (2008) 1.78
Meningitis caused by a serogroup W135 clone of the ET-37 complex of Neisseria meningitidis in West Africa. Trop Med Int Health (1998) 1.78
Natural infection with influenza A (H3N2). The development, persistance and effect of antibodies to the surface antigens. J Hyg (Lond) (1976) 1.77
Clinicoepidemiological profile and predictors of severe illness in young infants (< 60 days) reporting to a hospital in North India. Indian Pediatr (2007) 1.72
Survey of the frequency of USH1 gene mutations in a cohort of Usher patients shows the importance of cadherin 23 and protocadherin 15 genes and establishes a detection rate of above 90%. J Med Genet (2006) 1.72
Detection of over 98% cystic fibrosis mutations in a Celtic population. Nat Genet (1992) 1.72
Specialist phototrophs, lithotrophs, and methylotrophs: a unity among a diversity of procaryotes? Bacteriol Rev (1977) 1.72
The spectrum of skin disorders in abdominal stoma patients. Br J Dermatol (2000) 1.69
Polymorphic hydroxylation of debrisoquine. Lancet (1977) 1.69
Laparoscopic donor nephrectomy. Br J Surg (2003) 1.69
Clinical predictors of hypoxaemia in Gambian children with acute lower respiratory tract infection: prospective cohort study. BMJ (1999) 1.68
Transverse-momentum and pseudorapidity distributions of charged hadrons in pp collisions at square root of s = 7 TeV. Phys Rev Lett (2010) 1.67
Regulation of binding properties of the nicotinic receptor protein by cholinergic ligands in membrane fragments from Torpedo marmorata. Proc Natl Acad Sci U S A (1975) 1.66
Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8. Gene Ther (2007) 1.66
Gene therapy progress and prospects: the eye. Gene Ther (2006) 1.66
Evaluation of the Duke criteria versus the Beth Israel criteria for the diagnosis of infective endocarditis. Clin Infect Dis (1995) 1.65
Targeted disruption of outer limiting membrane junctional proteins (Crb1 and ZO-1) increases integration of transplanted photoreceptor precursors into the adult wild-type and degenerating retina. Cell Transplant (2010) 1.64
Simultaneous islet-kidney vs pancreas-kidney transplantation in type 1 diabetes mellitus: a 5 year single centre follow-up. Diabetologia (2007) 1.62
The bio-availability of fluoride from black tea. J Dent (2001) 1.62
Safety of retroviral gene marking with a truncated NGF receptor. Nat Med (2003) 1.60
The use of a radial haemolysis test for neuraminidase antibodies in the diagnosis of influenza A infection. J Hyg (Lond) (1983) 1.60
Search for supersymmetry using final states with one lepton, jets, and missing transverse momentum with the ATLAS detector in √s=7 TeV pp collisions. Phys Rev Lett (2011) 1.60