Human gene therapy. Clinical development

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