Human gene therapy

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Suppression of CNS APOE4 Expression by miRNAs Delivered by the S2 AAVrh.10 Capsid-modified AAV Vector. 2024
Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease.. 34. 2023
Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-labeled Adeno-associated Virus Capsids Administered to Cerebral Spinal Fluid. 2023
Assessment of Residual Full-Length SV40 Large T Antigen in Clinical-grade Adeno-associated Virus Vectors Produced in 293T Cells. 2023
Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia. 2023
Characterization of a Bioengineered AAV3B Capsid Variant with Enhanced Hepatocyte Tropism and Immune Evasion.. 34. 2023
Safety of Intravenous Administration of an AAV8 Vector Coding for an Oxidation Resistant Human α1-antitrypsin for the Treatment of α1-antitrypsin Deficiency. 2023
Identification of a Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friederichs’s Ataxia.. 605-615. 2023
Genetic Modification of the AAV5 Capsid with Lysine Residues Results in a Lung-tropic, Liver-detargeted Gene Transfer Vector. 2022
Safety of Direct Intraparenchymal AAVrh.10-Mediated Central Nervous System Gene Therapy for Metachromatic Leukodystrophy.. 32. 2021
Quantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates.. 31. 2020
Design and Rationale for First-in-Human Phase 1 Immunovirotherapy Clinical Trial of Oncolytic HSV G207 to Treat Malignant Pediatric Cerebellar Brain Tumors.. 31. 2020
Stress-Induced Mouse Model of the Cardiac Manifestations of Friedreich's Ataxia Corrected by AAV-mediated Gene Therapy.. 31. 2020
Single-Cell Transcriptome Analysis of Mouse Liver Cell-Specific Tropism and Transcriptional Dysregulation Following Intravenous Administration of AAVrh.10 Vectors.. 31. 2020
My Pathway to Gene Therapy.. 31. 2020
Systemic Adeno-Associated Virus-Mediated Gene Therapy Prevents the Multiorgan Disorders Associated with Aldehyde Dehydrogenase 2 Deficiency and Chronic Ethanol Ingestion.. 31. 2020
Anti-Phospho-Tau Gene Therapy for Chronic Traumatic Encephalopathy.. 31. 2019
Cytokine-induced killer cells engineered with exogenous T-cell receptors directed against melanoma antigens: enhanced efficacy of effector cells endowed with a double mechanism of tumor recognition.. 26. 2015
Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial.. 25. 2014
Epithelial-mesenchymal transition enhances response to oncolytic herpesviral therapy through nectin-1.. 25. 2014
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.. 25. 2014
Suppression of nicotine-induced pathophysiology by an adenovirus hexon-based antinicotine vaccine.. 24. 2013
Disrupted adenovirus-based vaccines against small addictive molecules circumvent anti-adenovirus immunity.. 24. 2012
Long-term follow-up assessment of a phase 1 trial of angiogenic gene therapy using direct intramyocardial administration of an adenoviral vector expressing the VEGF121 cDNA for the treatment of diffuse coronary artery disease.. 24. 2012
Recognition of glioma stem cells by genetically modified T cells targeting EGFRvIII and development of adoptive cell therapy for glioma.. 23. 2012
Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice.. 23. 2012
In vivo gene transfer strategies to achieve partial correction of von Willebrand disease.. 23. 2012
AAVrh.10-mediated expression of an anti-cocaine antibody mediates persistent passive immunization that suppresses cocaine-induced behavior.. 23. 2012
Persistent suppression of ocular neovascularization with intravitreal administration of AAVrh.10 coding for bevacizumab.. 22. 2011
When ethics constrains clinical research: trial design of control arms in "greater than minimal risk" pediatric trials.. 22. 2011
Coding polypurine hairpins cause target-induced cell death in breast cancer cells.. 22. 2011
Lentiviral-human heme oxygenase targeting endothelium improved vascular function in angiotensin II animal model of hypertension.. 22. 2011
Transduction of human antigen-presenting cells with integrase-defective lentiviral vector enables functional expansion of primed antigen-specific CD8(+) T cells.. 21. 2010
Protective immunity against a lethal respiratory Yersinia pestis challenge induced by V antigen or the F1 capsular antigen incorporated into adenovirus capsid.. 21. 2010
Genetic delivery of bevacizumab to suppress vascular endothelial growth factor-induced high-permeability pulmonary edema.. 20. 2009
Transient in vivo beta-globin production after lentiviral gene transfer to hematopoietic stem cells in the nonhuman primate.. 20. 2009
Functional cystic fibrosis transmembrane conductance regulator expression in cystic fibrosis airway epithelial cells by AAV6.2-mediated segmental trans-splicing.. 20. 2009
Chimeric receptor mRNA transfection as a tool to generate antineoplastic lymphocytes.. 20. 2009
Novel oncolytic agent GLV-1h68 is effective against malignant pleural mesothelioma.. 19. 2008
Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.. 19. 2008
Genetic delivery of the murine equivalent of bevacizumab (avastin), an anti-vascular endothelial growth factor monoclonal antibody, to suppress growth of human tumors in immunodeficient mice.. 19. 2008
Ablation of tumor-derived stem cells transplanted to the central nervous system by genetic modification of embryonic stem cells with a suicide gene.. 18. 2007
Multifactorial optimization of gammaretroviral gene transfer into human T lymphocytes for clinical application.. 18. 2007
Phase I, open-label, dose-escalating study of a genetically engineered herpes simplex virus, NV1020, in subjects with metastatic colorectal carcinoma to the liver.. 17. 2006
Functional assessment of the engraftment potential of gammaretrovirus-modified CD34+ cells, using a short serum-free transduction protocol.. 17. 2006
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.. 16. 2005
Protective immunity against alpha-cobratoxin following a single administration of a genetic vaccine encoding a non-toxic cobratoxin variant.. 16. 2005
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.. 15. 2004
Oncolytic herpes simplex virus-1 mutant expressing green fluorescent protein can detect and treat peritoneal cancer.. 15. 2004
A phase I vaccine safety and chemotherapy dose-finding trial of an allogeneic GM-CSF-secreting breast cancer vaccine given in a specifically timed sequence with immunomodulatory doses of cyclophosphamide and doxorubicin.. 15. 2004
Transgene expression after rep-mediated site-specific integration into chromosome 19.. 15. 2004
Protective immunity evoked against anthrax lethal toxin after a single intramuscular administration of an adenovirus-based vaccine encoding humanized protective antigen.. 14. 2003
Gene transfer to the pleural mesothelium as a strategy to deliver proteins to the lung parenchyma.. 13. 2002
Oncolytic herpesvirus effectively treats murine squamous cell carcinoma and spreads by natural lymphatics to treat sites of lymphatic metastases.. 13. 2002
Analysis of risk factors for local delivery of low- and intermediate-dose adenovirus gene transfer vectors to individuals with a spectrum of comorbid conditions.. 13. 2002
Safety of local delivery of low- and intermediate-dose adenovirus gene transfer vectors to individuals with a spectrum of morbid conditions.. 13. 2002
Intratumoral administration of low doses of an adenovirus vector encoding tumor necrosis factor alpha together with naive dendritic cells elicits significant suppression of tumor growth without toxicity.. 12. 2001
Prostate-specific antigen response and systemic T cell activation after in situ gene therapy in prostate cancer patients failing radiotherapy.. 12. 2001
Therapeutic effects of viral vector-mediated antiangiogenic gene transfer in malignant ascites.. 12. 2001
Adenovirus gene transfer vectors inhibit growth of lymphatic tumor metastases independent of a therapeutic transgene.. 12. 2001
A phase I/II dose escalation and activity study of intravenous injections of OCaP1 for subjects with refractory osteosarcoma metastatic to lung.. 12. 2001
Subthalamic GAD gene transfer in Parkinson disease patients who are candidates for deep brain stimulation.. 12. 2001
Cross-strain protection against clinical and laboratory strains of Pseudomonas aeruginosa mediated by dendritic cells genetically modified to express CD40 ligand and pulsed with specific strains of Pseudomonas aeruginosa.. 12. 2001
Immune consequences of intraocular administration of modified adenoviral vectors.. 12. 2001
Cytokine gene transfer enhances herpes oncolytic therapy in murine squamous cell carcinoma.. 12. 2001
Manipulation of the cytoplasmic and transmembrane domains alters cell surface levels of the coxsackie-adenovirus receptor and changes the efficiency of adenovirus infection.. 12. 2001
Human gene marker/therapy clinical protocols.. 11. 2000
Adenovirus vector-mediated gene transfer to regional lymph nodes.. 11. 2000
Coarse spray delivery to a localized region of the pulmonary airways for gene therapy.. 11. 2000
Dynein- and microtubule-mediated translocation of adenovirus serotype 5 occurs after endosomal lysis.. 11. 2000
Transient production of bone morphogenetic protein 2 by allogeneic transplanted transduced cells induces bone formation.. 11. 2000
Human gene marker/therapy clinical protocols.. 10. 1999
Cellular immune responses of healthy individuals to intradermal administration of an E1-E3- adenovirus gene transfer vector.. 10. 1999
Specific binding of the adenovirus capsid to the nuclear envelope.. 10. 1999
Anti-tumor immunity induced by in vivo adenovirus vector-mediated expression of CD40 ligand in tumor cells.. 10. 1999
Safety of direct myocardial administration of an adenovirus vector encoding vascular endothelial growth factor 121.. 10. 1999
In situ gene therapy for adenocarcinoma of the prostate: a phase I clinical trial.. 10. 1999
Suicide gene therapy for treatment of retinoblastoma in a murine model.. 10. 1999
Gene therapy for oxidant injury-related diseases: adenovirus-mediated transfer of superoxide dismutase and catalase cDNAs protects against hyperoxia but not against ischemia-reperfusion lung injury.. 9. 1998
Similarity of strain- and route-dependent murine responses to an adenovirus vector using the homologous thrombopoietin cDNA as the reporter genes.. 9. 1998
Regional suppression of tumor growth by in vivo transfer of a cDNA encoding a secreted form of the extracellular domain of the flt-1 vascular endothelial growth factor receptor.. 9. 1998
Efficient transfer of genes into murine cardiac grafts by Starburst polyamidoamine dendrimers.. 9. 1998
Fluorescent virions: dynamic tracking of the pathway of adenoviral gene transfer vectors in living cells.. 9. 1998
Retroviral transduction of human CD34+ umbilical cord blood progenitor cells with a mutated dihydrofolate reductase cDNA.. 9. 1998
Human gene marker/therapy clinical protocols.. 8. 1997
Promoter attenuation in gene therapy: interferon-gamma and tumor necrosis factor-alpha inhibit transgene expression.. 8. 1997
Role of alveolar macrophages in rapid elimination of adenovirus vectors administered to the epithelial surface of the respiratory tract.. 8. 1997
Adenovirus-mediated gene transfer of viral interleukin-10 inhibits the immune response to both alloantigen and adenoviral antigen.. 8. 1997
Cytotoxic T lymphocyte responses to proteins encoded by heterologous transgenes transferred in vivo by adenoviral vectors.. 8. 1997
Phase I study of direct administration of a replication deficient adenovirus vector containing the E. coli cytosine deaminase gene to metastatic colon carcinoma of the liver in association with the oral administration of the pro-drug 5-fluorocytosine.. 8. 1997
Delayed-type hypersensitivity response to high doses of adenoviral vectors.. 8. 1997
Regional angiogenesis induced in nonischemic tissue by an adenoviral vector expressing vascular endothelial growth factor.. 8. 1997
Circumvention of anti-adenovirus neutralizing immunity by administration of an adenoviral vector of an alternate serotype.. 8. 1997
Innate immune mechanisms dominate elimination of adenoviral vectors following in vivo administration.. 8. 1997
Human gene marker/therapy clinical protocols.. 7. 1996
Rapid production of interleukin-2-secreting tumor cells by herpes simplex virus-mediated gene transfer: implications for autologous vaccine production.. 7. 1996
Transfer of a gene encoding the anticandidal protein histatin 3 to salivary glands.. 7. 1996
Gene transfer of transforming growth factor-beta 1 prolongs murine cardiac allograft survival by inhibiting cell-mediated immunity.. 7. 1996
Regulatable promoters for use in gene therapy applications: modification of the 5'-flanking region of the CFTR gene with multiple cAMP response elements to support basal, low-level gene expression that can be upregulated by exogenous agents that raise intracellular levels of cAMP.. 7. 1996
Gene therapy in the United States: a five-year status report.. 7. 1996
Human gene marker/therapy clinical protocols.. 7. 1996
Regional delivery of an adenovirus vector containing the Escherichia coli cytosine deaminase gene to provide local activation of 5-fluorocytosine to suppress the growth of colon carcinoma metastatic to liver.. 7. 1996
Retrovirus-mediated gene transfer of 6-pyruvoyl-tetrahydropterin synthase corrects tetrahydrobiopterin deficiency in fibroblasts from hyperphenylalaninemic patients.. 7. 1996
Retroviral gene transfer in autologous bone marrow transplantation for adult acute leukemia.. 7. 1996
Arterial gene transfer for therapeutic angiogenesis in patients with peripheral artery disease.. 7. 1996
In vivo replication-deficient adenovirus vector-mediated transduction of the cytosine deaminase gene sensitizes glioma cells to 5-fluorocytosine.. 7. 1996
Prostate cancer gene therapy: herpes simplex virus thymidine kinase gene transduction followed by ganciclovir in mouse and human prostate cancer models.. 7. 1996
"Sero-switch" adenovirus-mediated in vivo gene transfer: circumvention of anti-adenovirus humoral immune defenses against repeat adenovirus vector administration by changing the adenovirus serotype.. 7. 1996
In vivo angiogenesis induced by recombinant adenovirus vectors coding either for secreted or nonsecreted forms of acidic fibroblast growth factor.. 6. 1995
Modification of nasal epithelial potential differences of individuals with cystic fibrosis consequent to local administration of a normal CFTR cDNA adenovirus gene transfer vector.. 6. 1995
In vivo adenovirus-mediated gene transfer of the Escherichia coli cytosine deaminase gene to human colon carcinoma-derived tumors induces chemosensitivity to 5-fluorocytosine.. 6. 1995
Evaluation of repeat administration of a replication deficient, recombinant adenovirus containing the normal cystic fibrosis transmembrane conductance regulator cDNA to the airways of individuals with cystic fibrosis.. 6. 1995
In vitro and in vivo transfer and expression of human surfactant SP-A- and SP-B-associated protein cDNAs mediated by replication-deficient, recombinant adenoviral vectors.. 6. 1995
Evaluation of the respiratory epithelium of normals and individuals with cystic fibrosis for the presence of adenovirus E1a sequences relevant to the use of E1a- adenovirus vectors for gene therapy for the respiratory manifestations of cystic fibrosis.. 5. 1994
Increasing expression of the normal human CFTR cDNA in cystic fibrosis epithelial cells results in a progressive increase in the level of CFTR protein expression, but a limit on the level of cAMP-stimulated chloride secretion.. 5. 1994
Acute responses of non-human primates to airway delivery of an adenovirus vector containing the human cystic fibrosis transmembrane conductance regulator cDNA.. 5. 1994
Direct in vivo gene transfer and expression in malignant cells using adenovirus vectors.. 5. 1994
Gene transfer to freshly isolated human respiratory epithelial cells in vitro using a replication-deficient adenovirus containing the human cystic fibrosis transmembrane conductance regulator cDNA.. 5. 1994
A pilot study of immunization with interleukin-2 secreting allogeneic HLA-A2 matched renal cell carcinoma cells in patients with advanced renal cell carcinoma.. 3. 1992
No retroviremia or pathology in long-term follow-up of monkeys exposed to a murine amphotropic retrovirus.. 2. 1991
Genetic Modification of the Lung Directed Toward Treatment of Human Disease. 2017
Adenovirus: the first effective in vivo gene delivery vector. 2014
Innate Immune Sensing of Adeno-Associated Virus Vectors. 2024
Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013. 2014
Gene therapy to stimulate angiogenesis to treat diffuse coronary artery disease. 2013
Therapeutic options for patients with severe beta-thalassemia: the need for globin gene therapy. 2007
Confronting the issues of therapeutic misconception, enrollment decisions, and personal motives in genetic medicine-based clinical research studies for fatal disorders. 2005
Adenovirus-based genetic vaccines for biodefense. 2005

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Human gene therapy Journal

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