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Human gene therapy. Clinical development
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Design and Rationale for First-in-Human Phase 1 Immunovirotherapy Clinical Trial of Oncolytic HSV G207 to Treat Malignant Pediatric Cerebellar Brain Tumors
. 31:1132-1139.
2020
Adeno-Associated Virus D-Sequence-Mediated Suppression of Expression of a Human Major Histocompatibility Class II Gene: Implications in the Development of Adeno-Associated Virus Vectors for Modulating Humoral Immune Response
. 31:565-574.
2020
Breathing New Life into TRAIL for Breast Cancer Therapy: Co-Delivery of pTRAIL and Complementary siRNAs Using Lipopolymers
. 30:1531-1546.
2019
Somatic Gene Editing of GUCY2D by AAV-CRISPR/Cas9 Alters Retinal Structure and Function in Mouse and Macaque
. 30:571-589.
2019
Improving the Transduction of Bone Marrow-Derived Cells with an Integrase-Defective Lentiviral Vector
. 29:44-59.
2018
Rationale and Design of a Phase 1 Clinical Trial to Evaluate HSV G207 Alone or with a Single Radiation Dose in Children with Progressive or Recurrent Malignant Supratentorial Brain Tumors
. 28:7-16.
2017
Rationale and Design of a Phase I Clinical Trial to Evaluate HSV G207 Alone or with a Single Radiation Dose in Children with Progressive or Recurrent Malignant Supratentorial Brain Tumors.
2017
Evaluation of Dose and Safety of AAV7m8 and AAV8BP2 in the Non-Human Primate Retina
. 28:154-167.
2017
Highly efficient delivery of adeno-associated viral vectors to the primate retina
. 27:580-597.
2016
Design of a Phase i Clinical Trial to Evaluate M032, a Genetically Engineered HSV-1 Expressing IL-12, in Patients with Recurrent/Progressive Glioblastoma Multiforme, Anaplastic Astrocytoma, or Gliosarcoma
. 27:69-78.
2016
Systemic Vascular Transduction by Capsid Mutant Adeno-Associated Virus after Intravenous Injection
. 26:767-776.
2015
My career path for developing gene therapy for blinding diseases: The importance of mentors, collaborators, and opportunities
. 25:663-670.
2014
Evaluation of the safety and biodistribution of M032, an attenuated herpes simplex virus type 1 expressing hIL-12, after intracerebral administration to Aotus Nonhuman Primates
. 25:16-27.
2014
Promising first steps in gene therapy for choroideremia
. 25:96-97.
2014
Transgene expression up to 7 years in nonhuman primates following hepatic transduction with helper-dependent adenoviral vectors
. 24:761-765.
2013
The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina.
. 23:1101-1115.
2012
A new generation of serotype chimeric infectivity-enhanced conditionally replicative adenovirals: The safety profile of Ad5/3-Δ24 in advance of a phase i clinical trial in ovarian cancer patients
. 22:821-828.
2011
Gene therapy in ophthalmology: Validation on cultured retinal cells and explants from postmortem human eyes
. 22:587-593.
2011
Generation of human induced pluripotent stem cells bearing an anti-HIV transgene by a lentiviral vector carrying an internal murine leukemia virus promoter
. 21:1556-1567.
2010
Genetic modification of adeno-associated viral vector type 2 capsid enhances gene transfer efficiency in polarized human airway epithelial cells
. 19:1407-1414.
2008
Human mesenchymal stem cells lack tumor tropism but enhance the antitumor activity of oncolytic adenoviruses in orthotopic lung and breast tumors
. 18:627-641.
2007
Novel recombinant adenoviral vector that targets the interleukin-13 receptor α2 chain permits effective gene transfer to malignant glioma
. 18:118-129.
2007
In vivo analysis of adenovirus-specific cytotoxic T lymphocyte response in mice deficient in CD28, Fas ligand, and perforin
. 17:669-682.
2006
In Vivo Analysis of Adenovirus-Specific Cytotoxic T Lymphocyte Response in Mice Deficient in CD28, Fas Ligand, and Perforin
. 0:060801084750020.
2006
An aye for eye gene therapy
. 17:177-179.
2006
Genomic stability of self-complementary adeno-associated virus 2 during early stages of transduction in mouse muscle in vivo
. 16:1047-1057.
2005
Partial redirection of transgenic human growth hormone secretion from rat salivary glands
. 16:571-583.
2005
Antibiotic-mediated chemoprotection enhances adaptation of E. coli PNP for herpes simplex virus-based glioma therapy
. 16:339-347.
2005
Efficient transduction of vascular endothelial cells with recombinant adeno-associated virus serotype 1 and 5 vectors
. 16:235-247.
2005
Long-term inducible gene expression in the eye via adeno-associated virus gene transfer in nonhuman primates
. 16:178-186.
2005
Intratumoral administration of a recombinant canarypox virus expressing interleukin 12 in patients with metastatic melanoma
. 16:91-100.
2005
Adenovirus-mediated delivery of catalase to retinal pigment epithelial cells protects neighboring photoreceptors from photo-oxidative stress
. 15:960-967.
2004
Augmentation of antitumor activity of a recombinant adeno-associated virus carcinoembryonic antigen vaccine with plasmid adjuvant
. 15:856-864.
2004
Osteogenic differentiation of recombinant adeno-associated virus 2-transduced murine mesenchymal stem cells and development of an immunocompetent mouse model for ex vivo osteoporosis gene therapy
. 15:1197-1206.
2004
Replicative oncolytic adenoviruses in multimodal cancer regimens
. 14:933-946.
2003
Efficient trans-splicing in the retina expands the utility of adeno-associated virus as a vector for gene therapy
. 14:37-44.
2003
Serum and ascites neutralizing antibodies in ovarian cancer patients treated with intraperitoneal adenoviral gene therapy
. 13:1505-1514.
2002
Multi-log cytotoxicity of carbocyclic 2′-deoxyguanosine in HSV-TK-expressing human tumor cells
. 13:543-551.
2002
Repetitive intrathecal injections of poliovirus replicons result in gene expression in neurons of the central nervous system without pathogenesis
. 12:1827-1841.
2001
Adeno-associated virus production of soluble tumor necrosis factor receptor neutralizes tumor necrosis factor α and reduces arthritis
. 11:2431-2442.
2000
A phase I-II trial to examine the toxicity of CMV- and EVB-specific cytotoxic T lymphocytes when used for prophylaxis against EVB and CMV disease in recipients of CD34-selected/T cell-depleted stem cell transplants
. 11:1453-1463.
2000
Prenatal gene transfer: Scientific, medical, and ethical issues: A report of the recombinant DNA advisory committee
. 11:1211-1229.
2000
Additional transduction events after subretinal readministration of recombinant adeno-associated virus
. 11:449-457.
2000
Using a tropism-modified adenoviral vector to circumvent inhibitory factors in ascites fluid
. 11:1657-1669.
2000
bcl-2 gene therapy exacerbates excitotoxicity
. 10:1715-1720.
1999
Nonspecific inflammation inhibits adenovirus-mediated pulmonary gene transfer and expression independent of specific acquired immune responses
. 9:2207-2222.
1998
Inhibition of tumor necrosis factor α decreases inflammation and prolongs adenovirus gene expression in lung and liver
. 9:1875-1884.
1998
Lung-specific expression of adenovirus E3-14.7K in transgenic mice attenuates adenoviral vector-mediated lung inflammation and enhances transgene expression
. 9:1885-1898.
1998
Repetitive adenovirus administration to the parotid gland: Role of immunological barriers and induction of oral tolerance
. 9:305-313.
1998
In vivo gene therapy of cancer with E. coli purine nucleoside phosphorylase
. 8:1637-1644.
1997
Lack of site-specific integration of the recombinant adeno-associated virus 2 genomes in human cells
. 8:275-284.
1997
A novel gene therapy strategy for elimination of prostate carcinoma cells from human bone marrow
. 8:157-170.
1997
Humoral response after administration of E1-deleted adenoviruses: Immune privilege of the subretinal space
. 7:1763-1769.
1996
Intraluminal water increases expression of plasmid DNA in rat lung
. 7:933-941.
1996
Informed consent to participate in a research study -- gene therapy for cystic fibrosis using cationic liposome mediated gene transfer: a phase I trial of safety and efficacy in the nasal airway.
. 5:1271-1277.
1994
Gene transfer into mammalian central nervous system using herpes virus vectors: Extended expression of bacterial lacZ in neurons using the neuron-specific enolase promoter
. 3:487-499.
1992
Identity
International Standard Serial Number (issn)
1043-0342
1946-6536
2324-8637
Electronic International Standard Serial Number (eissn)
1557-7422
1946-6544
2324-8645