| Year |
Title |
Altmetric |
| 2020
|
RNA Splicing Factor Mutations That Cause Retinitis Pigmentosa Result in Circadian Dysregulation.
Journal of Biological Rhythms.
35:72-83.
2020
|
|
| 2019
|
Safety of Same-Eye Subretinal Sequential Readministration of AAV2-hRPE65v2 in Non-human Primates.
Molecular Therapy - Methods and Clinical Development.
15:133-148.
2019
|
|
| 2019
|
AAV2.7m8 is a powerful viral vector for inner ear gene therapy.
Nature Communications.
10.
2019
|
|
| 2019
|
Utility of in Vitro Mutagenesis of RPE65 Protein for Verification of Mutational Pathogenicity before Gene Therapy.
JAMA Ophthalmology.
137:1381-1388.
2019
|
|
| 2019
|
CHOROIDEREMIA: Retinal Degeneration with an Unmet Need
2019
|
|
| 2019
|
Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation–Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials
2019
|
|
| 2019
|
AKT3 Gene Transfer Promotes Anabolic Reprogramming and Photoreceptor Neuroprotection in a Pre-clinical Model of Retinitis Pigmentosa.
Molecular Therapy.
27:1313-1326.
2019
|
|
| 2019
|
CRISPR Activation Enhances In Vitro Potency of AAV Vectors Driven by Tissue-Specific Promoters.
Molecular Therapy - Methods and Clinical Development.
13:380-389.
2019
|
|
| 2019
|
Identification of a novel pathogenic missense mutation in PRPF31 using whole exome sequencing: A case report.
British Journal of Ophthalmology.
103:761-767.
2019
|
|
| 2019
|
Detailed retinal phenotype of Boucher-Neuhäuser syndrome associated with mutations in PNPLA6 mimicking choroideremia.
Ophthalmic Genetics.
40:267-275.
2019
|
|
| 2019
|
Blinded by the light: A nonhuman primate model of achromatopsia.
Journal of Clinical Investigation.
129:513-515.
2019
|
|
| 2019
|
Comparative AAV-EGFP transgene expression using vector serotypes 1–9, 7M8, and 8b in human pluripotent stem cells, RPEs, and human and rat cortical neurons.
Stem Cells International.
2019.
2019
|
|
| 2019
|
Depicting brighter possibilities for treating blindness.
Science Translational Medicine.
11.
2019
|
|
| 2018
|
Prescreening whole exome sequencing results from patients with retinal degeneration for variants in genes associated with retinal degeneration.
Clinical Ophthalmology.
12:49-63.
2018
|
|
| 2018
|
Erratum to: An Autogenously Regulated Expression System for Gene Therapeutic Ocular Applications (Scientific Reports, (2015), 5, 1, (17105), 10.1038/srep17105).
Scientific Reports.
8.
2018
|
|
| 2018
|
RDH12 mutations cause a severe retinal degeneration with relatively spared rod function.
Investigative Ophthalmology and Visual Science.
59:5225-5236.
2018
|
|
| 2018
|
Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA.
Molecular Therapy : Nucleic Acids.
12:294-308.
2018
|
|
| 2018
|
Voretigene neparvovec-rzyl for the treatment of biallelic RPE65 mutation–associated retinal dystrophy.
Expert Opinion on Orphan Drugs.
6:457-464.
2018
|
|
| 2018
|
Comparing clinical perimetry and population receptive field measures in patients with choroideremia.
Investigative Ophthalmology and Visual Science.
59:3249-3258.
2018
|
|
| 2018
|
Amelioration of Neurosensory Structure and Function in Animal and Cellular Models of a Congenital Blindness.
Molecular Therapy.
26:1581-1593.
2018
|
|
| 2018
|
Novel mobility test to assess functional vision in patients with inherited retinal dystrophies.
Clinical and Experimental Ophthalmology.
46:247-259.
2018
|
|
| 2018
|
SIRT1 and NRF2 gene transfer mediate distinct neuroprotective effects upon retinal ganglion cell survival and function in experimental optic neuritis.
Investigative Ophthalmology and Visual Science.
59:1212-1220.
2018
|
|
| 2018
|
Use of induced pluripotent stem cell models to probe the pathogenesis of Choroideremia and to develop a potential treatment.
Stem Cell Research.
27:140-150.
2018
|
|
| 2018
|
On variants and disease-causing mutations: Case studies of a SEMA4A variant identified in inherited blindness.
Ophthalmic Genetics.
39:144-146.
2018
|
|
| 2018
|
Evidence for Amino Acid Sequence Differences among Proteins resembling the L-Chain Subunits of Immunoglobulins.
Journal of Immunology.
200:27-37.
2018
|
|
| 2017
|
Evaluating efficiencies of dual AAV approaches for retinal targeting.
Frontiers in Neuroscience.
11.
2017
|
|
| 2017
|
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
2017
|
|
| 2017
|
The Role of the Human Visual Cortex in Assessment of the Long-Term Durability of Retinal Gene Therapy in Follow-on RPE65 Clinical Trial Patients
2017
|
|
| 2017
|
Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward.
Molecular Therapy.
25:1076-1094.
2017
|
|
| 2017
|
Protocols for visually guided navigation assessment of efficacy of retina-directed cell or gene therapy in canines.
Frontiers in Neuroscience.
11.
2017
|
|
| 2017
|
Isolated maculopathy associated with biallelic CRB1 mutations.
Ophthalmic Genetics.
38:190-193.
2017
|
|
| 2017
|
Evaluation of Dose and Safety of AAV7m8 and AAV8BP2 in the Non-Human Primate Retina.
Human Gene Therapy.
28:154-167.
2017
|
|
| 2016
|
Institutional conflict of interest.
JAMA Ophthalmology.
134:1334-1335.
2016
|
|
| 2016
|
Gene Therapy and Stem Cell Transplantation in Retinal Disease: The New Frontier
2016
|
|
| 2016
|
Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial
2016
|
|
| 2016
|
In vitro and in vivo rescue of aberrant splicing in CEP290-associated LCA by antisense oligonucleotide delivery.
Human Molecular Genetics.
25:2552-2563.
2016
|
|
| 2016
|
Retinas in a Dish Peek into Inherited Retinal Degeneration.
Cell Stem Cell.
18:688-689.
2016
|
|
| 2016
|
The Thioredoxin Encoded by the Rod-Derived Cone Viability Factor Gene Protects Cone Photoreceptors Against Oxidative Stress.
Antioxidants and Redox Signaling.
24:909-923.
2016
|
|
| 2016
|
Corrigendum to “Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho–/– mouse” (Molecular Therapy - Methods and Clinical Development (2015) 2 (S2329050116300286) (10.1038/mtm.2015.16)).
Molecular Therapy - Methods and Clinical Development.
3:16032.
2016
|
|
| 2016
|
Does the site of anastomosis for esophagectomy affect long-term quality of life?.
Diseases of the Esophagus.
29:93-98.
2016
|
|
| 2015
|
An Autogenously Regulated Expression System for Gene Therapeutic Ocular Applications.
Scientific Reports.
5.
2015
|
|
| 2015
|
Plasticity of the human visual system after retinal gene therapy in patients with Leber's congenital amaurosis.
Science Translational Medicine.
7.
2015
|
|
| 2015
|
Erratum: AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models (PLOS ONE (2015) 10:6 (e0129982) (doi:10.1371/journal.pone.0129982)).
PLoS ONE.
10.
2015
|
|
| 2015
|
Basal exon skipping and genetic pleiotropy: A predictive model of disease pathogenesis.
Science Translational Medicine.
7.
2015
|
|
| 2015
|
Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho−/− mouse.
Molecular Therapy - Methods and Clinical Development.
2:15016.
2015
|
|
| 2015
|
Exploiting metabolic and antioxidant pathways to maintain vision in blinding disease.
Journal of Clinical Investigation.
125:1390-1392.
2015
|
|
| 2015
|
Neuromyelitis optica and multiple sclerosis: Seeing differences through optical coherence tomography
2015
|
|
| 2015
|
The status of RPE65 gene therapy trials: Safety and efficacy.
Cold Spring Harbor perspectives in medicine.
5.
2015
|
|
| 2015
|
Thioredoxin rod-derived cone viability factor protects against photooxidative retinal damage.
Free Radical Biology and Medicine.
81:22-29.
2015
|
|
| 2014
|
My career path for developing gene therapy for blinding diseases: The importance of mentors, collaborators, and opportunities.
Human Gene Therapy.
25:663-670.
2014
|
|
| 2014
|
Promising first steps in gene therapy for choroideremia.
Human Gene Therapy.
25:96-97.
2014
|
|
| 2014
|
fMRI of retina-originated phosphenes experienced by patients with leber congenital amaurosis.
PLoS ONE.
9.
2014
|
|
| 2014
|
Adeno-associated virus 8-mediated gene therapy for choroideremia: Preclinical studies in in vitro and in vivo models.
Journal of Gene Medicine.
16:122-130.
2014
|
|
| 2014
|
CEP290 and the primary cilium.
Advances in Experimental Medicine and Biology.
801:519-525.
2014
|
|
| 2014
|
Differential targeting of feline photoreceptors by recombinant adeno-associated viral vectors: Implications for preclinical gene therapy trials
2014
|
|
| 2014
|
Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno-associated virus capsid and promoter.
EMBO Molecular Medicine.
6:1175-1190.
2014
|
|
| 2014
|
High-resolution adaptive optics retinal imaging of cellular structure in choroideremia.
Investigative Ophthalmology and Visual Science.
55:6381-6397.
2014
|
|
| 2014
|
Novel mathematical algorithm for pupillometric data analysis.
Computer Methods and Programs in Biomedicine.
113:221-225.
2014
|
|
| 2014
|
Screening of a Large Cohort of Leber Congenital Amaurosis and Retinitis Pigmentosa Patients Identifies Novel LCA5 Mutations and New Genotype-Phenotype Correlations. [34, 11, 1537-1546, 10.1002/humu.22398].
Human Mutation.
35:150-150.
2014
|
|
| 2014
|
The Pex1-G844D mouse: A model for mild human Zellweger spectrum disorder.
Molecular Genetics and Metabolism.
111:522-532.
2014
|
|
| 2013
|
Disruption of CEP290 microtubule/membrane-binding domains causes retinal degeneration.
Journal of Clinical Investigation.
123:4525-4539.
2013
|
|
| 2013
|
Immunology of AAV-mediated gene transfer in the eye.
Frontiers in Immunology.
4.
2013
|
|
| 2013
|
Stem cells set their sights on retinitis pigmentosa: Skin cells from a patient with a form of inherited blindness have been reprogrammed into retinal cells and successfully transplanted into mice.
eLife.
2013.
2013
|
|
| 2013
|
Erratum: Progress in gene therapy for neurological disorders (Nature Reviews Neurology (2013) 9 (277-291) DOI: 10.1038/nrneurol.2013.56).
Nature Reviews Neurology.
9:298.
2013
|
|
| 2013
|
Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with leber congenital amaurosis type 2
2013
|
|
| 2013
|
AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models.
PLoS ONE.
8.
2013
|
|
| 2013
|
Seeing the light.
Science Translational Medicine.
5.
2013
|
|
| 2013
|
AAV9 Targets Cone Photoreceptors in the Nonhuman Primate Retina.
PLoS ONE.
8.
2013
|
|
| 2013
|
Progress in gene therapy for neurological disorders.
Nature Reviews Neurology.
9:277-291.
2013
|
|
| 2013
|
Screening of a large cohort of leber congenital amaurosis and retinitis pigmentosa patients identifies novel lca5 mutations and new genotype-phenotype correlations.
Human Mutation.
34:1537-1546.
2013
|
|
| 2012
|
Mutations in RPGR and RP2 account for 15% of males with simplex retinal degenerative disease.
Investigative Ophthalmology and Visual Science.
53:8232-8237.
2012
|
|
| 2012
|
The Bionic Retina: A Small Molecule with Big Potential for Visual Restoration.
Neuron.
75:185-187.
2012
|
|
| 2012
|
Pupillometric analysis for assessment of gene therapy in Leber Congenital Amaurosis patients.
BioMedical Engineering OnLine.
11.
2012
|
|
| 2012
|
The signalling role of the αvβ5-integrin can impact the efficacy of AAV in retinal gene therapy.
Pharmaceuticals.
5:447-459.
2012
|
|
| 2012
|
Nxnl2 splicing results in dual functions in neuronal cell survival and maintenance of cell integrity.
Human Molecular Genetics.
21:2298-2311.
2012
|
|
| 2012
|
Gene therapy: AAV2 gene therapy readministration in three adults with congenital blindness.
Science Translational Medicine.
4.
2012
|
|
| 2012
|
Antisense oligonucleotide (AON)-based therapy for leber congenital amaurosis caused by a frequent mutation in CEP290.
Molecular Therapy : Nucleic Acids.
1:e14.
2012
|
|
| 2011
|
Adeno-associated virus mediated gene therapy for retinal degenerative diseases.
Methods in Molecular Biology.
807:179-218.
2011
|
|
| 2011
|
Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey.
Science Translational Medicine.
3.
2011
|
|
| 2011
|
The evolution of retinal gene therapy: From DNA to FDA.
Retina Today.
55-58.
2011
|
|
| 2011
|
Switching on the lights: The use of optogenetics to advance retinal gene therapy.
Molecular Therapy.
19:1190-1192.
2011
|
|
| 2011
|
Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey.
Science Translational Medicine.
3.
2011
|
|
| 2011
|
Disruption of intraflagellar protein transport in photoreceptor cilia causes Leber congenital amaurosis in humans and mice.
Journal of Clinical Investigation.
121:2169-2180.
2011
|
|
| 2011
|
The human visual cortex responds to gene therapy-mediated recovery of retinal function.
Journal of Clinical Investigation.
121:2160-2168.
2011
|
|
| 2011
|
Gene therapy in ophthalmology: Validation on cultured retinal cells and explants from postmortem human eyes.
Human Gene Therapy.
22:587-593.
2011
|
|
| 2011
|
Erratum: Lighting a candle in the dark: Advances in genetics and gene therapy of recessive retinal dystrophies (Journal of Clinical Investigation (2010) 120, 9 (3042-3053) DOI: 10.1172/JCI42258).
Journal of Clinical Investigation.
121:456.
2011
|
|
| 2011
|
Erratum: The human visual cortex responds to gene therapy-mediated recovery of retinal function (The Journal of Clinical Investigation (2011) 121, 6, (2160-2168) DOI: 10.1172/JCI57377).
Journal of Clinical Investigation.
121:2945.
2011
|
|
| 2011
|
Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa.
Molecular Therapy.
19:642-649.
2011
|
|
| 2010
|
Lighting a candle in the dark: Advances in genetics and gene therapy of recessive retinal dystrophies.
Journal of Clinical Investigation.
120:3042-3053.
2010
|
|
| 2010
|
Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness.
Science Translational Medicine.
2.
2010
|
|
| 2010
|
Gene therapy for leber's congenital amaurosis is safe and effective through 1.5 years after vector administration.
Molecular Therapy.
18:643-650.
2010
|
|
| 2009
|
Gene therapy for color blindness.
New England Journal of Medicine.
361:2483.
2009
|
|
| 2009
|
Using the NAFX to measure the effectiveness over time of gene therapy in canine LCA.
Investigative Ophthalmology and Visual Science.
50:4685-4692.
2009
|
|
| 2009
|
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
2009
|
|
| 2009
|
Tie2Cre-mediated inactivation of plexinD1 results in congenital heart, vascular and skeletal defects.
Developmental Biology.
325:82-93.
2009
|
|
| 2008
|
Safety and efficacy of gene transfer for Leber's congenital amaurosis.
New England Journal of Medicine.
358:2240-2248.
2008
|
|
| 2008
|
Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice.
Journal of Clinical Investigation.
118:1955-1964.
2008
|
|
| 2008
|
Novel AAV serotypes for improved ocular gene transfer.
Journal of Gene Medicine.
10:375-382.
2008
|
|
| 2008
|
Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer.
Molecular Therapy.
16:458-465.
2008
|
|
| 2007
|
Accelerated mortality from hydrocephalus and pneumonia in mice with a combined deficiency of SPAG6 and SPAG16L reveals a functional interrelationship between the two central apparatus proteins.
Cytoskeleton.
64:360-376.
2007
|
|
| 2007
|
Retinal progenitor cells - Timing is everything.
New England Journal of Medicine.
356:1577-1579.
2007
|
|
| 2007
|
Gene transfer to ocular stem cells by early gestational intraamniotic injection of lentiviral vector.
Molecular Therapy.
15:579-587.
2007
|
|
| 2006
|
Retinal ganglion cell loss induced by acute optic neuritis in a relapsing model of multiple sclerosis
2006
|
|
| 2006
|
In Vivo Delivery of Recombinant Viruses to the Fetal Murine Cochlea: Transduction Characteristics and Long-Term Effects on Auditory Function.
Molecular Therapy.
14:328-335.
2006
|
|
| 2006
|
Eye movement recordings as an effectiveness indicator of gene therapy in RPE65-deficient canines: Implications for the ocular motor system.
Investigative Ophthalmology and Visual Science.
47:2865-2875.
2006
|
|
| 2006
|
Safety of Recombinant Adeno-Associated Virus Type 2-RPE65 Vector Delivered by Ocular Subretinal Injection.
Molecular Therapy.
13:1074-1084.
2006
|
|
| 2006
|
Deficiency of SPAG16L causes male infertility associated with impaired sperm motility.
Biology of Reproduction.
74:751-759.
2006
|
|
| 2006
|
An aye for eye gene therapy.
Human Gene Therapy.
17:177-179.
2006
|
|
| 2005
|
The distribution, concentration, and toxicity of enhanced green fluorescent protein in retinal cells after genomic or somatic (virus-mediated) gene transfer.
Molecular Vision.
11:1236-1245.
2005
|
|
| 2005
|
Spatial and temporal expression patterns of the choroideremia gene in the mouse retina.
Molecular Vision.
11:1052-1060.
2005
|
|
| 2005
|
ABCA4-associated retinal degenerations spare structure and function of the human parapapillary retina.
Investigative Ophthalmology and Visual Science.
46:4739-4746.
2005
|
|
| 2005
|
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.
Molecular Therapy.
12:1072-1082.
2005
|
|
| 2005
|
Herpes simplex virus type 1 glycoprotein E is required for axonal localization of capsid, tegument, and membrane glycoproteins.
Journal of Virology.
79:13362-13372.
2005
|
|
| 2005
|
Oxygen distribution and vascular injury in the mouse eye measured by phosphorescence-lifetime imaging.
Applied Optics.
44:5239-5248.
2005
|
|
| 2005
|
Dissecting the axoneme interactome: The mammalian orthologue of chlamydomonas PF6 interacts with sperm-associated antigen 6, the mammalian orthologue of Chlamydomonas PF16.
Molecular and Cellular Proteomics.
4:914-923.
2005
|
|
| 2005
|
Persistent fetal ocular vasculature in mice deficient in Bax and Bak.
JAMA Ophthalmology.
123:797-802.
2005
|
|
| 2005
|
Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success
2005
|
|
| 2005
|
Nonhuman primate models for diabetic ocular neovascularization using AAV2-mediated overexpression of vascular endothelial growth factor.
Diabetes.
54:1141-1149.
2005
|
|
| 2005
|
Disease expression in Usher syndrome caused by VLGR1 gene mutation (USH2C) and comparison with USH2A phenotype.
Investigative Ophthalmology and Visual Science.
46:734-743.
2005
|
|
| 2005
|
Long-term inducible gene expression in the eye via adeno-associated virus gene transfer in nonhuman primates.
Human Gene Therapy.
16:178-186.
2005
|
|
| 2004
|
Systemic rapamycin inhibits retinal and choroidal neovascularization in mice.
Molecular Vision.
10:964-972.
2004
|
|
| 2004
|
Adeno-associated viruses containing bFGF or BDNF are neuroprotective against excitotoxicity.
Current Eye Research.
29:379-386.
2004
|
|
| 2004
|
Effect of over-expression of pigment epithelium derived factor (PEDF) on developing retinal vasculature in the mouse.
Molecular Vision.
10:837-844.
2004
|
|
| 2004
|
Systemic but not intraocular Epo gene transfer protects the retina from light-and genetic-induced degeneration.
Molecular Therapy.
10:855-861.
2004
|
|
| 2004
|
Adenovirus-mediated delivery of catalase to retinal pigment epithelial cells protects neighboring photoreceptors from photo-oxidative stress.
Human Gene Therapy.
15:960-967.
2004
|
|
| 2004
|
Light damage induced changes in mouse retinal gene expression.
Experimental Eye Research.
79:239-247.
2004
|
|
| 2004
|
Impairment of the transient pupillary light reflex in Rpe65-/- mice and humans with leber congenital amaurosis.
Investigative Ophthalmology and Visual Science.
45:1259-1271.
2004
|
|
| 2004
|
Mutations in ABCA4 result in accumulation of lipofuscin before slowing of the retinoid cycle: A reappraisal of the human disease sequence.
Human Molecular Genetics.
13:525-534.
2004
|
|
| 2004
|
In utero gene therapy rescues vision in a murine model of congenital blindness.
Molecular Therapy.
9:182-188.
2004
|
|
| 2003
|
Increased expression of ceruloplasmin in the retina following photic injury.
Molecular Vision.
9:151-158.
2003
|
|
| 2003
|
Small interfering RNA (siRNA) targeting VEGF effectively inhibits ocular neovascularization in mouse model.
Molecular Vision.
9:210-216.
2003
|
|
| 2003
|
Proapoptotic Bcl-2 family members, Bax and Bak, are essential for developmental photoreceptor apoptosis.
Investigative Ophthalmology and Visual Science.
44:3598-3605.
2003
|
|
| 2003
|
Delivery of adeno-associated virus vectors to the fetal retina: Impact of viral capsid proteins on retinal neuronal progenitor transduction.
Journal of Virology.
77:7957-7963.
2003
|
|
| 2003
|
Immune response following intraocular delivery of recombinant viral vectors
2003
|
|
| 2003
|
Adeno-associated virus encoding green fluorescent protein as a label for retinal pigment epithelium.
Investigative Ophthalmology and Visual Science.
44:772-780.
2003
|
|
| 2003
|
Efficient trans-splicing in the retina expands the utility of adeno-associated virus as a vector for gene therapy.
Human Gene Therapy.
14:37-44.
2003
|
|
| 2003
|
Gene therapy and animal models for retinal disease..
Developments in Ophthalmology.
37:188-198.
2003
|
|
| 2003
|
Gene therapy for Leber congenital amaurosis.
Novartis Foundation symposium.
255:195-202.
2003
|
|
| 2003
|
Gene therapy for ocular neovascularization: A cure in sight.
Current Opinion in Genetics and Development.
13:317-322.
2003
|
|
| 2002
|
Pigment epithelium-derived factor expression in the developing mouse eye.
Molecular Vision.
8:449-454.
2002
|
|
| 2002
|
Lateral connectivity and contextual interactions in macaque primary visual cortex.
Neuron.
36:739-750.
2002
|
|
| 2002
|
Phenotypic rescue after adeno-associated virus-mediated delivery of 4-sulfatase to the retinal pigment epithelium of feline mucopolysaccharidosis VI.
Journal of Gene Medicine.
4:613-621.
2002
|
|
| 2002
|
Inhibition of retinal neovascularization by intraocular viral-mediated delivery of anti-angiogenic agents.
Molecular Therapy.
6:490-494.
2002
|
|
| 2002
|
Pharmacological regulation of protein expression from adeno-associated viral vectors in the eye.
Molecular Therapy.
6:238-242.
2002
|
|
| 2002
|
A deviant immune response to viral proteins and transgene product is generated on subretinal administration of adenovirus and adeno-associated virus.
Molecular Therapy.
5:125-132.
2002
|
|
| 2002
|
Macular pigment and lutein supplementation in choroideremia.
Experimental Eye Research.
74:371-381.
2002
|
|
| 2002
|
Novel mutation in the TIMP3 gene causes Sorsby fundus dystrophy.
JAMA Ophthalmology.
120:376-379.
2002
|
|
| 2001
|
Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: The retina as a model.
Human Molecular Genetics.
10:3075-3081.
2001
|
|
| 2001
|
Localization of pigment epithelium derived factor (PEDF) in developing and adult human ocular tissues.
Molecular Vision.
7:154-163.
2001
|
|
| 2001
|
Gene therapy and retinitis pigmentosa: Advances and future challenges.
BioEssays.
23:662-668.
2001
|
|
| 2001
|
Strain-dependent anterior segment neovascularization following intravitreal gene transfer of basic fibroblast growth factor (bFGF).
Journal of Gene Medicine.
3:252-259.
2001
|
|
| 2001
|
Melatonin delays photoreceptor degeneration in the rds/rds mouse.
NeuroReport.
12:1011-1014.
2001
|
|
| 2001
|
Pharmacologically regulated gene expression in the retina following transduction with viral vectors
2001
|
|
| 2001
|
AAV-mediated delivery of ciliary neurotrophic factor prolongs photoreceptor survival in the rhodopsin knockout mouse.
Molecular Therapy.
3:241-248.
2001
|
|
| 2001
|
Gene therapy restores vision in a canine model of childhood blindness.
Nature Genetics.
28:92-95.
2001
|
|
| 2001
|
Long-term protection of retinal structure but not function using rAAV.CNTF in animal models of retinitis pigmentosa.
Molecular Therapy.
4:461-472.
2001
|
|
| 2000
|
Gene therapy for retinitis pigmentosa.
Current Opinion in Molecular Therapeutics.
2:420-425.
2000
|
|
| 2000
|
Additional transduction events after subretinal readministration of recombinant adeno-associated virus.
Human Gene Therapy.
11:449-457.
2000
|
|
| 2000
|
Application of adenoviral vectors. Analysis of eye development..
Methods in Molecular Biology.
135:525-535.
2000
|
|
| 2000
|
Disease expression of RP1 mutations causing autosomal dominant retinitis pigmentosa.
Investigative Ophthalmology and Visual Science.
41:1898-1908.
2000
|
|
| 2000
|
Gene therapy for ocular disease.
Molecular Therapy.
1:501-505.
2000
|
|
| 2000
|
Mutation of a nuclear receptor gene, NR2E3, causes enhanced S cone syndrome, a disorder of retinal cell fate.
Nature Genetics.
24:127-131.
2000
|
|
| 1999
|
Basic fibroblast growth factor induces a transformed phenotype in normal human melanocytes.
Oncogene.
18:6469-6476.
1999
|
|
| 1999
|
Infection with adeno-associated virus may protect against excitotoxicity.
NeuroReport.
10:2887-2890.
1999
|
|
| 1999
|
Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina
1999
|
|
| 1999
|
Persistent transgene product in retina, optic nerve and brain after intraocular injection of rAAV.
Vision Research.
39:2545-2553.
1999
|
|
| 1999
|
bcl-2 gene therapy exacerbates excitotoxicity.
Human Gene Therapy.
10:1715-1720.
1999
|
|
| 1998
|
Temporal and spatial regulation of gene expression mediated by the promoter for the human tissue inhibitor of metalloproteinases-3 (TIMP-3)- encoding gene.
Developmental Dynamics.
211:228-237.
1998
|
|
| 1998
|
Adenovirus-mediated delivery of rhodopsin-promoted bcl-2 results in a delay in photoreceptor cell death in the rd/rd mouse
1998
|
|
| 1997
|
Adenovirus mediated genetic therapy for hereditary retinal degeneration.
Investigative Ophthalmology and Visual Science.
38.
1997
|
|
| 1997
|
Adenovirus-mediated delivery of bcl-2 in conjunction with pde prolongs the delay in photoreceptor cell death in the rd/rd mouse.
Investigative Ophthalmology and Visual Science.
38.
1997
|
|
| 1997
|
Development of cataracts in a line of mice transgenic for human rhodopsin-promoted LacZ.
Investigative Ophthalmology and Visual Science.
38.
1997
|
|
| 1997
|
Real-time, noninvasive in vivo assessment of adeno-associated virus- mediated retinal transduction.
Investigative Ophthalmology and Visual Science.
38:2857-2863.
1997
|
|
| 1997
|
Cell-mediated immune response and stability of intraocular transgene expression after adenovirus-mediated delivery.
Investigative Ophthalmology and Visual Science.
38:2224-2233.
1997
|
|
| 1997
|
A carboxy-terminal truncation of 99 amino acids resulting from a novel mutation (Arg555-->stop) in the CHM gene leads to choroideremia..
Experimental Eye Research.
64:487-490.
1997
|
|
| 1997
|
Spectrum of mutations in the RPGR gene that are identified in 20% of families with X-linked retinitis pigmentosa.
American Journal of Human Genetics.
61:1287-1292.
1997
|
|
| 1996
|
Humoral response after administration of E1-deleted adenoviruses: Immune privilege of the subretinal space.
Human Gene Therapy.
7:1763-1769.
1996
|
|
| 1996
|
Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy.
Nature Medicine.
2:649-654.
1996
|
|
| 1996
|
Humoral response after administration of E1-deleted adenoviruses: Immune privilege of the subretinal space.
Investigative Ophthalmology and Visual Science.
37.
1996
|
|
| 1996
|
Preferential rod and cone photoreceptor abnormalities in heterozygotes with point mutations in the RDS gene..
Experimental Eye Research.
63:603-608.
1996
|
|
| 1995
|
Sequence analysis of the 5.34-kb 5′ flanking region of the human rhodopsin-encoding gene.
Gene.
167:317-320.
1995
|
|
| 1995
|
In vivo gene transfer into murine corneal endothelial and trabecular meshwork cells.
Investigative Ophthalmology and Visual Science.
36:2211-2215.
1995
|
|
| 1994
|
Adenovirus vector-mediated in vivo gene transfer into adult murine retina.
Investigative Ophthalmology and Visual Science.
35:2535-2542.
1994
|
|
| 1994
|
Training the future internal medicine subspecialist.
American Journal of Medicine.
96:559-561.
1994
|
|
| 1993
|
The Affliction of Internal Medicine.
New England Journal of Medicine.
329:212.
1993
|
|
| 1993
|
Generating more generalists: An agenda of renewal for internal medicine.
Annals of Internal Medicine.
119:1125-1129.
1993
|
|
| 1993
|
Interactions of cell-surface galactosyltransferase with immunoglobulins.
Molecular Immunology.
30:265-275.
1993
|
|
| 1993
|
Interactions of galactosyltransferase with serum and secretory immunoglobulins and their component chains.
Molecular Immunology.
30:277-286.
1993
|
|
| 1993
|
Medical informatics and the need for general internists [6].
Annals of Internal Medicine.
118:750-751.
1993
|
|
| 1993
|
United we stand.
Annals of Internal Medicine.
118:903-904.
1993
|
|
| 1992
|
A locus control region adjacent to the human red and green visual pigment genes.
Neuron.
9:429-440.
1992
|
|
| 1992
|
Activism in academic internal medicine.
Annals of Internal Medicine.
117:259-260.
1992
|
|
| 1992
|
Development of a didactic curriculum in science related to internal medicine.
Annals of Internal Medicine.
116:1088-1090.
1992
|
|
| 1992
|
General internal medicine and general internists: Recognizing a national need.
Annals of Internal Medicine.
117:778-779.
1992
|
|
| 1991
|
Unusual topography of bovine rhodopsin promoter-IacZ fusion gene expression in transgenic mouse retinas.
Neuron.
6:187-199.
1991
|
|
| 1989
|
Analysis of the spectrin binding domains of globin.
Biochemical and Biophysical Research Communications.
164:894-902.
1989
|
|
| 1988
|
Conversion of the Beckman liquid phase sequencer to a gas-liquid phase sequencer.
Analytical Biochemistry.
175:39-51.
1988
|
|
| 1987
|
Heterogeneity of binding of monoclonal IgA to staphylococcal protein A is related to the IgA polymerization state.
Immunology Letters.
16:11-14.
1987
|
|
| 1987
|
Interactions between surface molecules and the cytoskeleton in the lymphocyte.
1987
|
|
| 1987
|
The infectious etiology of chronic rheumatic diseases.
Seminars in Arthritis and Rheumatism.
17:1-23.
1987
|
|
| 1986
|
Biochemical characterization of Fcγ receptors of mouse DCH-5 cell line.
Immunobiology.
172:81-91.
1986
|
|
| 1986
|
Interleukin 1-like factor produced by a hybrid of an adherent mouse spleen cell and a thymoma cell.
Immunology Letters.
13:143-149.
1986
|
|
| 1986
|
Purification of spfctrin and its subunits by high-performance liquid chromatography
1986
|
|
| 1986
|
Simple device for high-performance liquid chromatographic separation on microbore columns
1986
|
|
| 1986
|
The stoichiometry of J chain in human secretory dimeric IgA.
Molecular Immunology.
23:541-544.
1986
|
|
| 1985
|
Secondary structure of the immunoglobulin J chain. (circular dichroism/hydrophobicity profile/tertiary structure/antiparallel β-barrel)
1985
|
|
| 1985
|
A modified system for thiazolinone conversion to thiohydantoin derivatives and their separation by high-pressure liquid chromatography.
Analytical Biochemistry.
150:457-462.
1985
|
|
| 1985
|
Isolation, characterization, and amino-terminal amino acid sequence analysis of human neutrophil elastase from normal donors.
Analytical Biochemistry.
149:153-162.
1985
|
|
| 1984
|
Use of mini-octadecylsilane ultrasphere column in high-pressure liquid chromatography for protein structural studies.
Analytical Biochemistry.
137:256-260.
1984
|
|
| 1984
|
Amino Acid Sequence of Human D of the Alternative Complement Pathway.
Biochemistry.
23:2482-2486.
1984
|
|
| 1984
|
Identification of phenylthiohydantoin derivatives of substituted amino acids encountered during amono acid sequence analysis by high-performance liquid chromatography
1984
|
|
| 1984
|
Partial characterisation of the high and low molecular weight forms of P388D1‐derived interleukin 1.
Journal of Cellular Biochemistry.
26:65-73.
1984
|
|
| 1983
|
A comparison of fluorescamine and o-phthaldialdehyde as effective blocking reagents in protein sequence analyses by the Beckman sequencer.
Analytical Biochemistry.
131:337-340.
1983
|
|
| 1983
|
American rheumatology: markings.
Arthritis and Rheumatism.
26:548-552.
1983
|
|
| 1983
|
Amino-terminal amino acid sequence of p10, the fifth major gag polypeptide of avian sarcoma and leukemia viruses.
Journal of Virology.
45:885-888.
1983
|
|
| 1983
|
Exclusive Use of High Pressure Liquid Chromatography for The Determination of the Complete Amino Acid Sequence of The 12K Fragment of Avian Sarcoma Virus Structural Protein p27
1983
|
|
| 1982
|
Buffer electrofocusing of Interleukin I
1982
|
|
| 1982
|
Selective dansylation of M protein within intact influenza virions.
Journal of Virology.
44:871-876.
1982
|
|
| 1982
|
The H-2kk antigen: Isolation using monoclonal immunoadsorbent chromatography and sequence analysis without radioisotopes.
Molecular Immunology.
19:1-11.
1982
|
|
| 1981
|
Interleukin 1: Production by P388D1 cells attached to microcarrier beads.
Journal of Immunological Methods.
46:197-204.
1981
|
|
| 1981
|
Purification and characterization of the gag gene products of avian-type C retroviruses by high-pressure liquid chromatography.
Analytical Biochemistry.
112:128-134.
1981
|
|
| 1981
|
Use of fluorescamine as an effective blocking reagent to reduce the background in protein sequence analyses by the beckman automated sequencer.
Analytical Biochemistry.
112:158-162.
1981
|
|
| 1981
|
An improved procedure for high-sensitivity microsequencing: Use of aminoethyl aminopropyl glass beads in the beckman sequencer and the Ultrasphere ODS column for PTH amino acid identification.
Analytical Biochemistry.
110:355-359.
1981
|
|
| 1981
|
Reverse phase ion pair high performance liquid chromatography of viral tryptic glycopeptides
1981
|
|
| 1980
|
Monoclonal and allospecific H-2K(k) antisera precipitate a high molecular weight protein.
Journal of Immunology.
125:2295-2298.
1980
|
|
| 1980
|
Alignment of the peptides derived from acid-catalyzed cleavage of an aspartylprolyl bond in the major internal structural polypeptide of avian retroviruses.
Journal of Biological Chemistry.
255:6962-6965.
1980
|
|
| 1980
|
A simple modification on the vacuum system of the Beckman automated sequencer to improve the efficiency of edman degradation.
Analytical Biochemistry.
102:35-38.
1980
|
|
| 1980
|
Partial amino acid sequence of human factor D:homology with serine proteases.
1980
|
|
| 1980
|
Separation of influenza hemagglutinin tryptic glycopeptides by ion-pair reverse-phase high-performance liquid chromatography (HPLC)
1980
|
|
| 1979
|
Reverse phase ion‐pair chromatography of viral glycopeptides
1979
|
|
| 1979
|
Structure of the membrane protein of influenza virus. I. Isolation and characterization of cyanogen bromide cleavage products.
Journal of Virology.
30:759-766.
1979
|
|
| 1978
|
Computer-Assisted high-pressure liquid chromatography of radio-labelled phenylthiohydantoin amino acids
1978
|
|
| 1978
|
Methanol solvent system for rapid analysis of phenylthiohydantoin amino acids by high-pressure liquid chromatography
1978
|
|
| 1978
|
Amino-terminal amino acid sequence of the major structural polypeptides of avian retroviruses: Sequence homology between reticuloendotheliosis virus p30 and p30s of mammalian retroviruses
1978
|
|
| 1978
|
Quantitative yields of underivatized peptides by HPLC
1978
|
|
| 1978
|
The infectious etiology of rheumatoid arthritis..
Arthritis and Rheumatism.
21:531-538.
1978
|
|
| 1978
|
The molecular structure and interrelationships of murine T lymphoid cell-surface antigens..
Contemporary topics in molecular immunology.
7:283-317.
1978
|
|
| 1977
|
Primary Structure of Human J Chain: Alignment of Peptides from Chemical and Enzymic Hydrolyses.
Biochemistry.
16:3507-3513.
1977
|
|
| 1977
|
Primary Structure of Human J Chain: Isolation and Characterization of Tryptic and Chymotryptic Peptides of Human J Chain.
Biochemistry.
16:3501-3507.
1977
|
|
| 1977
|
A Preliminary Study Using Electron Capture Detection of Pentafluorophenylthiohydantoin Amino Acid Derivatives
1977
|
|
| 1977
|
Structural analysis of the peptides derived from specific acid catalyzed hydrolysis at aspartylprolyl peptide bonds in human J chain.
Journal of Immunology.
118:67-70.
1977
|
|
| 1976
|
Sequence analysis of human J chain. Amino terminal location of a disulfide bond linking the immunoglobulin heavy chain.
Biochemical and Biophysical Research Communications.
73:92-97.
1976
|
|
| 1976
|
A comparative analysis of the C1 binding ability of fragments derived from complement fixing and non complement fixing IgM proteins.
Journal of Clinical Investigation.
58:16-21.
1976
|
|
| 1976
|
Antibodies to human native and denatured collagens in synovial fluids of patients with rheumatoid arthritis.
Clinical immunology and immunopathology.
6:209-212.
1976
|
|
| 1976
|
Isolation and properties of the CNBr peptides of influenza viral membrane protein.
The FASEB Journal.
35.
1976
|
|
| 1976
|
Quantitative Isolation of Mouse Thymus Leukemia Antigen, TL
1976
|
|
| 1975
|
Subfragments of papain solubilized TL antigen.
Journal of Immunology.
115:1013-1017.
1975
|
|
| 1975
|
Purification of Murine Thymus Leukemia Antigen (TL). A Quantitative Assessment of Limited Proteolysis.
Biochemistry.
14:4792-4796.
1975
|
|
| 1975
|
C1 fixation and classical complement pathway activation by a fragment of the Cμ4 domain of IgM.
Journal of Experimental Medicine.
142:1322-1326.
1975
|
|
| 1975
|
The primary structure of J chain: sequence analyses of the major proteolytic fragments.
The FASEB Journal.
34:4160.
1975
|
|
| 1973
|
Rheumatoid arthritis: an immune complex disease?
1973
|
|
| 1973
|
Characterization of a teleost immunoglobulin: The immune macroglobulin from the channel catfish, Ictalurus punctatus.
Comparative Biochemistry and Physiology - B: Comparative Biochemistry.
46.
1973
|
|
| 1973
|
Isolation and characterization of the glycopeptides from a human immunoglobulin M..
Journal of Immunology.
110:840-847.
1973
|
|
| 1973
|
Isolation of f(c)5μ and fabμ fragments of human igm.
European Journal of Immunology.
3:415-419.
1973
|
|
| 1972
|
The carbohydrate composition of immunoglobulins from diverse species of vertebrates..
Journal of Immunology.
109:371-381.
1972
|
|
| 1972
|
Purification and Characterization of Two Classes of Immunoglobulins from the Marine Toad, Bufo marinus.
Biochemistry.
11:2751-2759.
1972
|
|
| 1972
|
Studies of the component chains of human IgM by citraconylation.
Biochimica et Biophysica Acta - Protein Structure.
263:339-350.
1972
|
|
| 1971
|
Isolation and characterization of the immune macroglobulin from the paddlefish, Polyodon spathula..
Journal of Biological Chemistry.
246:6760-6769.
1971
|
|
| 1971
|
Degradation of human IgM by pepsin: characterization of a high molecular weight fragment..
Journal of Immunology.
107:870-880.
1971
|
|
| 1971
|
Isolation and physico-chemical characterization of the "IgM-like" immunologlobulin from the stingray Dasyatis americana..
Journal of Immunology.
107:782-793.
1971
|
|
| 1971
|
The carbohydrate composition of immunoglobulins G
1971
|
|
| 1971
|
Phylogeny of Immunoglobulins. Characterization of a 14S Immunoglobuim from the Gar, Lepisosteus osseus.
Biochemistry.
10:2028-2036.
1971
|
|
| 1971
|
Oxidative sulfitolysis of human IgM..
Journal of Immunology.
106:1136-1138.
1971
|
|
| 1971
|
Tetrameric immune macroglobulins in three orders of bony fishes.
1971
|
|
| 1970
|
Biological sciences: N-Terminal sequences of immunoglobulin heavy and light chains from three species of lower vertebrates.
Nature.
228:991-992.
1970
|
|
| 1970
|
Studies of maleylated chains of human IgM..
Journal of Immunology.
105:846-855.
1970
|
|
| 1970
|
Antibodies to ribosomal ribonucleic acid (rRNA) in patients with systemic lupus erythematosus (SLE)..
Immunology.
19:439-442.
1970
|
|
| 1970
|
Release of Fab- and Fc-like fragments from human IgM by reductive cleavage..
Journal of Immunology.
104:518-521.
1970
|
|
| 1970
|
Antibodies to Homologous RNA in the Rabbit Following Stimulation By Exogenous RNA (34922)
1970
|
|
| 1969
|
Physical and chemical characterization of an oyster hemagglutinin..
Journal of Biological Chemistry.
244:4128-4135.
1969
|
|
| 1969
|
Immunological capabilities of the oyster Crassostrea virginica.
Comparative Biochemistry and Physiology.
29:149-160.
1969
|
|
| 1969
|
Management of rheumatoid arthritis.
Southern Medical Journal.
62:307-314.
1969
|
|
| 1969
|
Possible Usefulness of Azathioprine (Imuran) In Severe Rheumatoid Arthritis.
The Journal of clinical pharmacology and the journal of new drugs.
9:251-258.
1969
|
|
| 1969
|
Studies on the structural variation of the H chain of immunoglobulin M(IgM).
Archives of Biochemistry and Biophysics.
131:551-560.
1969
|
|
| 1968
|
The Amino-Terminal Sequence of the Heavy Chain of Human Immunoglobulin M.
Biochemistry.
7:3340-3344.
1968
|
|
| 1967
|
[36] Paper chromatography and electrophoresis; special procedure for peptide maps.
Methods in Enzymology.
11:330-339.
1967
|
|
| 1966
|
Concluding remarks: the regulation of hemoglobin and immunoglobulin synthesis..
Journal of Cellular Physiology.
67.
1966
|
|
| 1966
|
Concluding remarks: The regulation of hemoglobin and immunoglobulin synthesis.
Journal of Cellular Physiology.
67:207-215.
1966
|
|
| 1965
|
The molecular basis of antibody formation: a paradox.
1965
|
|
| 1964
|
The relationship of the three dimensional conformation of proteins to their antigenic specificity.
Medicine.
43:305-314.
1964
|
|
| 1963
|
Studies on antigen conformation during antibody purification..
Journal of Biological Chemistry.
238:1362-1366.
1963
|
|
| 1962
|
Polarization of fluorescence as a measure of antigen-antibody interaction.
1962
|
|
