Kaminsky, Stephen M.

Publications

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selected publications

Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease. Human gene therapy. 2025 Academic Article GET IT
Vectorized Human Antibody-Mediated Anti-Eosinophil Gene Therapy. Human gene therapy. 2024 Academic Article GET IT
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
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
Times cited: 3
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
Times cited: 2
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
Times cited: 7
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
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
Automated Retinal Layer Segmentation in CLN2-Associated Disease: Commercially Available Software Characterizing a Progressive Maculopathy. Translational vision science & technology. 2021 Academic Article GET IT
Times cited: 4
Safety of Direct Intraparenchymal AAVrh.10-Mediated Central Nervous System Gene Therapy for Metachromatic Leukodystrophy. Human gene therapy. 2021 Academic Article GET IT
Times cited: 24
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 GET IT
Times cited: 51
Quantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates. Human gene therapy. 2020 Academic Article GET IT
Times cited: 24
Cocaine vaccine dAd5GNE protects against moderate daily and high-dose "binge" cocaine use. PloS one. 2020 Academic Article GET IT
Gene therapy for alpha 1-antitrypsin deficiency with an oxidant-resistant human alpha 1-antitrypsin. JCI insight. 2020 Academic Article GET IT
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 GET IT
Times cited: 2
Symmetric Age Association of Retinal Degeneration in Patients with CLN2-Associated Batten Disease. Ophthalmology. Retina. 2020 Academic Article GET IT
Times cited: 18
Anti-Phospho-Tau Gene Therapy for Chronic Traumatic Encephalopathy. Human gene therapy. 2019 Academic Article GET IT
Times cited: 4
Accurate Quantification and Characterization of Adeno-Associated Viral Vectors. Frontiers in microbiology. 2019 Academic Article GET IT
Times cited: 89
Gene therapy for C1 esterase inhibitor deficiency in a Murine Model of Hereditary angioedema. Allergy. 2019 Academic Article GET IT
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 GET IT
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 GET IT
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 GET IT
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 GET IT
Times cited: 117
Radioiodinated Capsids Facilitate In Vivo Non-Invasive Tracking of Adeno-Associated Gene Transfer Vectors. Scientific reports. 2017 Academic Article GET IT
Times cited: 20
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 GET IT
Times cited: 55
Gene therapy for metachromatic leukodystrophy. Journal of neuroscience research. 2016 Review GET IT
Times cited: 73
Anti-Epidermal Growth Factor Receptor Gene Therapy for Glioblastoma. PloS one. 2016 Academic Article GET IT
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 GET IT
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 GET IT
Times cited: 44
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 GET IT
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 GET IT
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 GET IT
Times cited: 20
Evaluation of compounded bevacizumab prepared for intravitreal injection. JAMA ophthalmology. 2015 Academic Article GET IT
Times cited: 32
Genetic modification of neurons to express bevacizumab for local anti-angiogenesis treatment of glioblastoma. Cancer gene therapy. 2014 Academic Article GET IT
Times cited: 19
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 GET IT
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 GET IT
Times cited: 54
AAV-mediated persistent bevacizumab therapy suppresses tumor growth of ovarian cancer. Gynecologic oncology. 2014 Academic Article GET IT
Times cited: 25
"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 GET IT
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 GET IT
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 GET IT
Times cited: 39
Gene therapy to stimulate angiogenesis to treat diffuse coronary artery disease. Human gene therapy. 2013 Review GET IT
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 GET IT
Times cited: 40
Suppression of nicotine-induced pathophysiology by an adenovirus hexon-based antinicotine vaccine. Human gene therapy. 2013 Academic Article GET IT
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 GET IT
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 GET IT
Times cited: 9
Disrupted adenovirus-based vaccines against small addictive molecules circumvent anti-adenovirus immunity. Human gene therapy. 2012 Academic Article GET IT
Times cited: 21
Assessment of disease severity in late infantile neuronal ceroid lipofuscinosis using multiparametric MR imaging. AJNR. American journal of neuroradiology. 2012 Academic Article GET IT
Times cited: 18
AAV-directed persistent expression of a gene encoding anti-nicotine antibody for smoking cessation. Science translational medicine. 2012 Academic Article GET IT
Times cited: 48
In vivo gene transfer strategies to achieve partial correction of von Willebrand disease. Human gene therapy. 2012 Academic Article GET IT
Times cited: 18
AAVrh.10-mediated expression of an anti-cocaine antibody mediates persistent passive immunization that suppresses cocaine-induced behavior. Human gene therapy. 2012 Academic Article GET IT
Times cited: 39
Persistent suppression of ocular neovascularization with intravitreal administration of AAVrh.10 coding for bevacizumab. Human gene therapy. 2011 Academic Article GET IT
Times cited: 21
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 GET IT
Times cited: 75
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 GET IT
Times cited: 57
Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations. Journal of neurosurgery. Pediatrics. 2010 Academic Article GET IT
Times cited: 61
Broad and potent neutralizing antibodies from an African donor reveal a new HIV-1 vaccine target. Science (New York, N.Y.). 2009 Academic Article GET IT
Times cited: 1482
HIV-1 rational vaccine design: molecular details of b12-gp120 complex structure. Expert review of vaccines. 2007 Article GET IT
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 GET IT
Times cited: 119
Transfer of the AQP1 cDNA for the correction of radiation-induced salivary hypofunction. Biochimica et biophysica acta. 2005 Information Resource GET IT
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 GET IT
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 GET IT
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 GET IT
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 GET IT
Times cited: 100
Feasibility of gene therapy for late neuronal ceroid lipofuscinosis. Archives of neurology. 2001 Information Resource GET IT
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 GET IT
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 GET IT
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 GET IT
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 GET IT
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 GET IT
Times cited: 21

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Research

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Funding awarded

missing activity Principal Investigator 2020 - 2024

Gene Therapy for Alpha 1-Antitrypsin Deficiency awarded by National Heart, Lung, & Blood Institute Co-Investigator 2024 - 2025
Rapid Noninvasive Whole-body Imaging of AAV Gene Transfer Vectors awarded by National Institute of Biomedical Imaging & Bioengineering Co-Investigator 2019 - 2025
missing activity Co-Investigator 2019 - 2024
missing activity Co-Investigator 2019 - 2024

Phase IA/IB Study of AAVrh.10hFXN Therapy to Treat the Cardiomyopathy of Friedreich''s Ataxia awarded by National Heart, Lung, & Blood Institute Co-Principal Investigator 2020 - 2025

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Teaching

teaching overview

Lecturer: MOLECULAR BIOLOGY AND GENETICS IN CLINICAL/TRANSLATIONAL RESEARCH

Background

education and training

B.S., Cornell University 79
Ph.D., Yale University 1989
M.Phil., Yale University 1984

Contact

full name

Stephen M. Kaminsky

primary email

smkamins@med.cornell.edu

webpage

Dept of Genetic Medicine

VIVO Weill Cornell Medical College

Stephen M. Kaminsky Professor of Research in Genetic Medicine

Publications

selected publications

Research

Funding awarded

Teaching

teaching overview

Background

education and training

Contact

full name

primary email

webpage

Phone

Publications

selected publications

Research

Funding awarded

Teaching

teaching overview

Background

education and training

Contact

full name

primary email

webpage