selected publications
- Expression and processing of mature human frataxin after gene therapy in mice. Scientific reports. 2024 Academic Article GET IT
- Expression and processing of mature human frataxin after gene therapy in mice. 2023 GET IT
- Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease. Human gene therapy. 2023 Academic Article GET IT
- Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-labeled Adeno-associated Virus Capsids Administered to Cerebral Spinal Fluid. Human gene therapy. 2023 Academic Article GET IT
- Preclinical Safety and Biodistribution Assessment of Ad-KCNH2-G628S Administered via Atrial Painting in New Zealand White Rabbits. Basic & clinical pharmacology & toxicology. 2023 Academic Article GET IT
- Assessment of Residual Full-Length SV40 Large T Antigen in Clinical-grade Adeno-associated Virus Vectors Produced in 293T Cells. Human gene therapy. 2023 Academic Article GET IT
- Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia. Human gene therapy. 2023 Academic Article GET IT
- Safety of Intravenous Administration of an AAV8 Vector Coding for an Oxidation Resistant Human α1-antitrypsin for the Treatment of α1-antitrypsin Deficiency. Human gene therapy. 2023 Academic Article GET IT
- Genetic Modification of the AAV5 Capsid with Lysine Residues Results in a Lung-tropic, Liver-detargeted Gene Transfer Vector. Human gene therapy. 2022 Academic Article GET IT
- Long-term functional correction of cystathionine β-synthase deficiency in mice by adeno-associated viral gene therapy. Journal of inherited metabolic disease. 2021 Academic Article GET IT
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Automated Retinal Layer Segmentation in CLN2-Associated Disease: Commercially Available Software Characterizing a Progressive Maculopathy.
Translational vision science & technology.
2021
Academic Article
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Times cited: 3 -
Safety of Direct Intraparenchymal AAVrh.10-Mediated Central Nervous System Gene Therapy for Metachromatic Leukodystrophy.
Human gene therapy.
2021
Academic Article
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Times cited: 23 -
Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2.
Science translational medicine.
2020
Academic Article
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Times cited: 46 -
Quantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates.
Human gene therapy.
2020
Academic Article
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Times cited: 23 - Cocaine vaccine dAd5GNE protects against moderate daily and high-dose "binge" cocaine use. PloS one. 2020 Academic Article GET IT
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Gene therapy for alpha 1-antitrypsin deficiency with an oxidant-resistant human alpha 1-antitrypsin.
JCI insight.
2020
Academic Article
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Times cited: 13 -
Stress-Induced Mouse Model of the Cardiac Manifestations of Friedreich's Ataxia Corrected by AAV-mediated Gene Therapy.
Human gene therapy.
2020
Academic Article
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Times cited: 2 -
Symmetric Age Association of Retinal Degeneration in Patients with CLN2-Associated Batten Disease.
Ophthalmology. Retina.
2020
Academic Article
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Times cited: 16 -
Anti-Phospho-Tau Gene Therapy for Chronic Traumatic Encephalopathy.
Human gene therapy.
2019
Academic Article
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Times cited: 4 -
Accurate Quantification and Characterization of Adeno-Associated Viral Vectors.
Frontiers in microbiology.
2019
Academic Article
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Times cited: 83 -
Gene therapy for C1 esterase inhibitor deficiency in a Murine Model of Hereditary angioedema.
Allergy.
2019
Academic Article
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Times cited: 36 -
Untargeted Metabolite Profiling of Cerebrospinal Fluid Uncovers Biomarkers for Severity of Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2, Batten Disease).
Scientific reports.
2018
Academic Article
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Times cited: 20 -
In Vivo Potency Assay for Adeno-Associated Virus-Based Gene Therapy Vectors Using AAVrh.10 as an Example.
Human gene therapy methods.
2018
Academic Article
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Times cited: 8 -
Attenuation of the Niemann-Pick type C2 disease phenotype by intracisternal administration of an AAVrh.10 vector expressing Npc2.
Experimental neurology.
2018
Academic Article
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Times cited: 10 -
AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease.
Human gene therapy. Clinical development.
2018
Academic Article
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Times cited: 113 -
Radioiodinated Capsids Facilitate In Vivo Non-Invasive Tracking of Adeno-Associated Gene Transfer Vectors.
Scientific reports.
2017
Academic Article
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Times cited: 17 -
Vectored Intracerebral Immunization with the Anti-Tau Monoclonal Antibody PHF1 Markedly Reduces Tau Pathology in Mutant Tau Transgenic Mice.
The Journal of neuroscience : the official journal of the Society for Neuroscience.
2016
Academic Article
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Times cited: 55 -
Gene therapy for metachromatic leukodystrophy.
Journal of neuroscience research.
2016
Review
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Times cited: 69 -
Anti-Epidermal Growth Factor Receptor Gene Therapy for Glioblastoma.
PloS one.
2016
Academic Article
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Times cited: 18 -
Efficacy of an adenovirus-based anti-cocaine vaccine to reduce cocaine self-administration and reacqusition using a choice procedure in rhesus macaques.
Pharmacology, biochemistry, and behavior.
2016
Academic Article
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Times cited: 24 -
In situ reprogramming to transdifferentiate fibroblasts into cardiomyocytes using adenoviral vectors: Implications for clinical myocardial regeneration.
The Journal of thoracic and cardiovascular surgery.
2016
Academic Article
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Times cited: 42 -
Anti-hIgE gene therapy of peanut-induced anaphylaxis in a humanized murine model of peanut allergy.
The Journal of allergy and clinical immunology.
2016
Academic Article
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Times cited: 18 -
Intracerebral adeno-associated virus gene delivery of apolipoprotein E2 markedly reduces brain amyloid pathology in Alzheimer's disease mouse models.
Neurobiology of aging.
2016
Academic Article
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Times cited: 31 -
Brain Region-Specific Degeneration with Disease Progression in Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2 Disease).
AJNR. American journal of neuroradiology.
2016
Academic Article
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Times cited: 19 -
Evaluation of compounded bevacizumab prepared for intravitreal injection.
JAMA ophthalmology.
2015
Academic Article
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Times cited: 32 -
Genetic modification of neurons to express bevacizumab for local anti-angiogenesis treatment of glioblastoma.
Cancer gene therapy.
2014
Academic Article
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Times cited: 18 -
Phase I/II study of intrapleural administration of a serotype rh.10 replication-deficient adeno-associated virus gene transfer vector expressing the human α1-antitrypsin cDNA to individuals with α1-antitrypsin deficiency.
Human gene therapy. Clinical development.
2014
Academic Article
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Times cited: 15 -
Comparative efficacy and safety of multiple routes of direct CNS administration of adeno-associated virus gene transfer vector serotype rh.10 expressing the human arylsulfatase A cDNA to nonhuman primates.
Human gene therapy. Clinical development.
2014
Academic Article
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Times cited: 54 -
AAV-mediated persistent bevacizumab therapy suppresses tumor growth of ovarian cancer.
Gynecologic oncology.
2014
Academic Article
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Times cited: 24 -
"Triplet" polycistronic vectors encoding Gata4, Mef2c, and Tbx5 enhances postinfarct ventricular functional improvement compared with singlet vectors.
The Journal of thoracic and cardiovascular surgery.
2014
Academic Article
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Times cited: 34 -
Fate of systemically administered cocaine in nonhuman primates treated with the dAd5GNE anticocaine vaccine.
Human gene therapy. Clinical development.
2014
Academic Article
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Times cited: 29 -
Intrapleural administration of an AAVrh.10 vector coding for human α1-antitrypsin for the treatment of α1-antitrypsin deficiency.
Human gene therapy. Clinical development.
2013
Academic Article
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Times cited: 39 -
Gene therapy to stimulate angiogenesis to treat diffuse coronary artery disease.
Human gene therapy.
2013
Review
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Times cited: 19 -
Spectrum of ocular manifestations in CLN2-associated batten (Jansky-Bielschowsky) disease correlate with advancing age and deteriorating neurological function.
PloS one.
2013
Academic Article
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Times cited: 37 -
Suppression of nicotine-induced pathophysiology by an adenovirus hexon-based antinicotine vaccine.
Human gene therapy.
2013
Academic Article
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Times cited: 16 -
Adenovirus capsid-based anti-cocaine vaccine prevents cocaine from binding to the nonhuman primate CNS dopamine transporter.
Neuropsychopharmacology : official publication of the American College of Neuropsychopharmacology.
2013
Academic Article
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Times cited: 41 -
Safety of direct cardiac administration of AdVEGF-All6A+, a replication-deficient adenovirus vector cDNA/genomic hybrid expressing all three major isoforms of human vascular endothelial growth factor, to the ischemic myocardium of rats.
Human gene therapy. Clinical development.
2013
Academic Article
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Times cited: 9 -
Disrupted adenovirus-based vaccines against small addictive molecules circumvent anti-adenovirus immunity.
Human gene therapy.
2012
Academic Article
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Times cited: 21 -
Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis.
Human gene therapy methods.
2012
Academic Article
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Times cited: 84 -
Assessment of disease severity in late infantile neuronal ceroid lipofuscinosis using multiparametric MR imaging.
AJNR. American journal of neuroradiology.
2012
Academic Article
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Times cited: 17 -
AAV-directed persistent expression of a gene encoding anti-nicotine antibody for smoking cessation.
Science translational medicine.
2012
Academic Article
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Times cited: 48 -
In vivo gene transfer strategies to achieve partial correction of von Willebrand disease.
Human gene therapy.
2012
Academic Article
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Times cited: 17 -
AAVrh.10-mediated expression of an anti-cocaine antibody mediates persistent passive immunization that suppresses cocaine-induced behavior.
Human gene therapy.
2012
Academic Article
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Times cited: 39 -
Persistent suppression of ocular neovascularization with intravitreal administration of AAVrh.10 coding for bevacizumab.
Human gene therapy.
2011
Academic Article
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Times cited: 20 -
Novel cocaine vaccine linked to a disrupted adenovirus gene transfer vector blocks cocaine psychostimulant and reinforcing effects.
Neuropsychopharmacology : official publication of the American College of Neuropsychopharmacology.
2011
Academic Article
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Times cited: 74 -
Cocaine analog coupled to disrupted adenovirus: a vaccine strategy to evoke high-titer immunity against addictive drugs.
Molecular therapy : the journal of the American Society of Gene Therapy.
2011
Academic Article
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Times cited: 56 -
Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations.
Journal of neurosurgery. Pediatrics.
2010
Academic Article
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Times cited: 59 -
Broad and potent neutralizing antibodies from an African donor reveal a new HIV-1 vaccine target.
Science (New York, N.Y.).
2009
Academic Article
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Times cited: 1468 - HIV-1 rational vaccine design: molecular details of b12-gp120 complex structure. Expert review of vaccines. 2007 Article GET IT
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Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis.
The Journal of neuroscience : the official journal of the Society for Neuroscience.
2006
Academic Article
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Times cited: 117 -
Transfer of the AQP1 cDNA for the correction of radiation-induced salivary hypofunction.
Biochimica et biophysica acta.
2005
Information Resource
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Times cited: 44 -
Safety of direct administration of AAV2(CU)hCLN2, a candidate treatment for the central nervous system manifestations of late infantile neuronal ceroid lipofuscinosis, to the brain of rats and nonhuman primates.
Human gene therapy.
2005
Academic Article
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Times cited: 39 -
AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL.
Gene therapy.
2005
Academic Article
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Times cited: 58 -
Confronting the issues of therapeutic misconception, enrollment decisions, and personal motives in genetic medicine-based clinical research studies for fatal disorders.
Human gene therapy.
2005
Review
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Times cited: 27 -
Clinical protocol. Administration of a replication-deficient adeno-associated virus gene transfer vector expressing the human CLN2 cDNA to the brain of children with late infantile neuronal ceroid lipofuscinosis.
Human gene therapy.
2004
Academic Article
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Times cited: 100 -
Feasibility of gene therapy for late neuronal ceroid lipofuscinosis.
Archives of neurology.
2001
Information Resource
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Times cited: 24 -
Evaluation of a lipopeptide immunogen as a therapeutic in HIV type 1-seropositive individuals.
AIDS research and human retroviruses.
2000
Academic Article
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Times cited: 33 -
Na(+)-I- symport activity is present in membrane vesicles from thyrotropin-deprived non-I(-)-transporting cultured thyroid cells.
Proceedings of the National Academy of Sciences of the United States of America.
1994
Academic Article
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Times cited: 97 -
Differences in hydrogen exchange behavior between the oxidized and reduced forms of Escherichia coli thioredoxin.
Protein science : a publication of the Protein Society.
1992
Academic Article
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Times cited: 24 -
Reduction of thioredoxin significantly decreases its partial specific volume and adiabatic compressibility.
Protein science : a publication of the Protein Society.
1992
Academic Article
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Times cited: 23 -
Inhibition of the Na+/I- symporter by harmaline and 3-amino-1-methyl-5H-pyrido(4,3-b)indole acetate in thyroid cells and membrane vesicles.
European journal of biochemistry.
1991
Academic Article
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Times cited: 21