selected publications
- AAVrh.10 Delivery of Novel APOE2-Christchurch Variant Suppresses Amyloid and Tau Pathology in Alzheimer's Disease Mice. Molecular therapy : the journal of the American Society of Gene Therapy. 2024 Academic Article GET IT
- 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
- 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
- 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: 47 -
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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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 -
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 -
Disease characteristics and progression in patients with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease: an observational cohort study.
The Lancet. Child & adolescent health.
2018
Academic Article
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Times cited: 93 -
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: 18 -
Genetic Modification of the Lung Directed Toward Treatment of Human Disease.
Human gene therapy.
2017
Information Resource
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Times cited: 23 -
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: 70 -
Anti-Epidermal Growth Factor Receptor Gene Therapy for Glioblastoma.
PloS one.
2016
Academic Article
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Times cited: 18 -
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 -
Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates.
Human gene therapy. Clinical development.
2015
Academic Article
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Times cited: 48 -
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: 19 -
Intra-arterial delivery of AAV vectors to the mouse brain after mannitol mediated blood brain barrier disruption.
Journal of controlled release : official journal of the Controlled Release Society.
2014
Academic Article
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Times cited: 74 -
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 -
Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene.
Human gene therapy.
2014
Academic Article
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Times cited: 34 -
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 -
Human iPSC models of neuronal ceroid lipofuscinosis capture distinct effects of TPP1 and CLN3 mutations on the endocytic pathway.
Human molecular genetics.
2013
Academic Article
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Times cited: 110 -
Cannulation of the internal carotid artery in mice: a novel technique for intra-arterial delivery of therapeutics.
Journal of neuroscience methods.
2013
Academic Article
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Times cited: 8 -
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 -
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 -
Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice.
Human gene therapy.
2012
Academic Article
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Times cited: 74 -
Novel therapies, high-risk pediatric research, and the prospect of benefit: learning from the ethical disagreements.
Molecular therapy : the journal of the American Society of Gene Therapy.
2012
Academic Article
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Times cited: 2 -
When ethics constrains clinical research: trial design of control arms in "greater than minimal risk" pediatric trials.
Human gene therapy.
2011
Academic Article
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Times cited: 12 -
Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations.
Journal of neurosurgery. Pediatrics.
2010
Academic Article
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Times cited: 59 -
Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.
Human gene therapy.
2008
Academic Article
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Times cited: 295 -
Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis.
Experimental neurology.
2008
Academic Article
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Times cited: 60 -
Neurological deterioration in late infantile neuronal ceroid lipofuscinosis.
Neurology.
2007
Academic Article
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Times cited: 69 -
Assessing disease severity in late infantile neuronal ceroid lipofuscinosis using quantitative MR diffusion-weighted imaging.
AJNR. American journal of neuroradiology.
2007
Academic Article
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Times cited: 25 -
Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector.
Molecular therapy : the journal of the American Society of Gene Therapy.
2006
Academic Article
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Times cited: 125 -
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: 118 -
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 -
Domain interactions in protein tyrosine kinase Csk.
Biochemistry.
1999
Academic Article
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Times cited: 59 -
Expressed protein ligation: a general method for protein engineering.
Proceedings of the National Academy of Sciences of the United States of America.
1998
Academic Article
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Times cited: 1036 -
Peptide and protein phosphorylation by protein tyrosine kinase Csk: insights into specificity and mechanism.
Biochemistry.
1998
Academic Article
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Times cited: 76