| Year |
Title |
Altmetric |
| 2019
|
AAV2.7m8 is a powerful viral vector for inner ear gene therapy.
Nature Communications.
10.
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 & 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 & 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 & 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.
Lancet.
390:849-860.
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.
Ophthalmology: Journal of The American Academy of Ophthalmology.
124:873-883.
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.
Ophthalmology: Journal of The American Academy of Ophthalmology.
123:S98-S106.
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.
Lancet.
388:661-672.
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
|
|
| 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
|
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.
Gene Therapy.
21:913-920.
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 & 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.
Ophthalmology: Journal of The American Academy of Ophthalmology.
120:1283-1291.
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 & 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 & 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.
Lancet.
374:1597-1605.
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.
Cell Motility and the 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.
Multiple Sclerosis.
12:526-532.
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 & 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 & 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.
Archives of Ophthalmology -New Series-.
123:797-802.
2005
|
|
| 2005
|
Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success.
Proceedings of the National Academy of Sciences.
102:6177-6182.
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 & 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 & 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 & 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.
Gene Therapy.
10:977-982.
2003
|
|
| 2003
|
Adeno-associated virus encoding green fluorescent protein as a label for retinal pigment epithelium.
Investigative Ophthalmology & 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.
Archives of Ophthalmology -New Series-.
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.
Gene Therapy.
8:442-446.
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
|
Disease expression of RP1 mutations causing autosomal dominant retinitis pigmentosa.
Investigative Ophthalmology & Visual Science.
41:1898-1908.
2000
|
|
| 2000
|
Additional transduction events after subretinal readministration of recombinant adeno-associated virus.
Human Gene Therapy.
11:449-457.
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
|
|
| 2000
|
Application of adenoviral vectors. Analysis of eye development..
Methods in Molecular Biology.
135:525-535.
2000
|
|
| 2000
|
Gene therapy for ocular disease.
Molecular Therapy.
1:501-505.
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.
Proceedings of the National Academy of Sciences.
96:9920-9925.
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
|
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| 1998
|
Adenovirus-mediated delivery of rhodopsin-promoted bcl-2 results in a delay in photoreceptor cell death in the rd/rd mouse.
Gene Therapy.
5:1156-1164.
1998
|
|
| 1997
|
Adenovirus mediated genetic therapy for hereditary retinal degeneration.
Investigative Ophthalmology & 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 & Visual Science.
38.
1997
|
|
| 1997
|
Development of cataracts in a line of mice transgenic for human rhodopsin-promoted LacZ.
Investigative Ophthalmology & Visual Science.
38.
1997
|
|
| 1997
|
Real-time, noninvasive in vivo assessment of adeno-associated virus- mediated retinal transduction.
Investigative Ophthalmology & Visual Science.
38:2857-2863.
1997
|
|
| 1997
|
Cell-mediated immune response and stability of intraocular transgene expression after adenovirus-mediated delivery.
Investigative Ophthalmology & 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 & 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 & Visual Science.
36:2211-2215.
1995
|
|
| 1994
|
Adenovirus vector-mediated in vivo gene transfer into adult murine retina.
Investigative Ophthalmology & 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..
Transactions- American Clinical and Climatological Association.
99:119-125.
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.
Chromatographic reviews.
369:159-164.
1986
|
|
| 1986
|
Simple device for high-performance liquid chromatographic separation on microbore columns.
Chromatographic reviews.
370:508-512.
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).
Proceedings of the National Academy of Sciences.
82:5905-5909.
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.
Chromatographic reviews.
314:467-470.
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
|
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
|
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
|
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.
Journal of liquid chromatography.
6:249-258.
1983
|
|
| 1982
|
Selective dansylation of M protein within intact influenza virions.
Journal of Virology.
44:871-876.
1982
|
|
| 1982
|
Buffer electrofocusing of Interleukin I.
Bioscience Reports.
2:241-246.
1982
|
|
| 1982
|
The H-2k k antigen: Isolation using monoclonal immunoadsorbent chromatography and sequence analysis without radioisotopes.
Molecular Immunology.
19:1-11.
1982
|
|
| 1981
|
Interleukin 1: Production by P388D 1 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.
Journal of liquid chromatography.
4:587-598.
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..
Proceedings of the National Academy of Sciences.
77:1116-1119.
1980
|
|
| 1980
|
Separation of influenza hemagglutinin tryptic glycopeptides by ion-pair reverse-phase high-performance liquid chromatography (HPLC).
Journal of Biochemical and Biophysical Methods.
3:61-63.
1980
|
|
| 1979
|
Reverse phase ion‐pair chromatography of viral glycopeptides.
Hrc -Heidelberg-.
2:149-150.
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
|
The molecular structure and interrelationships of murine T lymphoid cell-surface antigens..
Contemporary topics in molecular immunology.
7:283-317.
1978
|
|
| 1978
|
Computer-Assisted high-pressure liquid chromatography of radio-labelled phenylthiohydantoin amino acids.
Chromatographic reviews.
156:35-41.
1978
|
|
| 1978
|
Methanol solvent system for rapid analysis of phenylthiohydantoin amino acids by high-pressure liquid chromatography.
Chromatographic reviews.
148:532-535.
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.
Proceedings of the National Academy of Sciences.
75:2708-2712.
1978
|
|
| 1978
|
Quantitative yields of underivatized peptides by HPLC.
Hrc -Heidelberg-.
1:177-178.
1978
|
|
| 1978
|
The infectious etiology of rheumatoid arthritis..
Arthritis and Rheumatism.
21:531-538.
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.
Proceedings of the Society for Experimental Biology and Medicine.
155:287-289.
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.
Federation proceedings.
35.
1976
|
|
| 1976
|
Quantitative Isolation of Mouse Thymus Leukemia Antigen, TL.
Tissue Antigens.
7:158-164.
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.
Federation proceedings.
34:4160.
1975
|
|
| 1973
|
Rheumatoid arthritis: an immune complex disease?.
Geriatrics -Minnesota-.
28:84-91.
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) 5 μ 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.
BBA - General Subjects.
237:442-449.
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..
Proceedings of the National Academy of Sciences.
68:107-111.
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).
Proceedings of the Society for Experimental Biology and Medicine.
134:968-970.
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
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|
| 1965
|
The molecular basis of antibody formation: a paradox..
Proceedings of the National Academy of Sciences.
54:864-869.
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..
Proceedings of the National Academy of Sciences.
48:1935-1942.
1962
|
|
